Attracting Clinical Trials to the UK

ACT-BT can lead the way

31st August 2026

ACT-BT gives us the road map - but where are the roads?

Finding the right clinical trial is only half the problem. What happens when the trial the UK needs isn’t here?

Yesterday, BTNL looked at ACT-BT - Access to Clinical Trials for Brain Tumours

It’s a brilliant concept.

For years, patients and families have been expected to navigate the clinical-trials system largely for themselves: searching databases, deciphering eligibility criteria, discovering that a trial they thought looked promising closed six months ago, or trying to persuade an already overstretched consultant to investigate something happening at another hospital.

ACT-BT changes that.

Adults with primary brain tumours anywhere in the UK can now be referred by their healthcare team to a specialist panel which meets weekly, examines their individual circumstances and identifies clinical trials for which they may be suitable.

That is an important step forward.

But it raises another question.

What happens when ACT-BT finds the right kind of trial - but the trial isn’t in the UK?

Or worse:

What happens when the treatment looks sufficiently promising to justify a trial, UK patients need that trial, UK hospitals are capable of conducting it - but nobody has brought the trial here?

ACT-BT may have solved part of the clinical-trial access problem.

But it cannot, by itself, solve the clinical-trial supply problem.

Safusidenib: a case in point

Many of you will know that safusidenib is an experimental oral, brain-penetrant inhibitor of mutant IDH1 being developed by Nuvation Bio for IDH1-mutant glioma.

First of all, let’s have a quick re-cap on where we are with this drug.

• In July, Nuvation reported updated results from 27 Japanese patients with previously untreated Grade 2 IDH1-mutant glioma. The confirmed objective response rate was 51.9%, while 79.1% of patients remained progression-free at three years.¹

• These are encouraging results, but they come from a small, non-randomised study and need confirmation in much larger trials.

• That confirmation is now being sought.

• On 20 August, the US FDA granted safusidenib Fast Track Designation for IDH1-mutant glioma.²

• Nuvation is simultaneously expanding the clinical programme across several stages of the disease.

And this is where things become interesting from a UK patient’s perspective.

SIGMA: global - but not currently in the UK

The pivotal SIGMA Phase III trial, NCT05303519, is testing safusidenib in higher-risk IDH1-mutant astrocytoma, including Grade 4 disease, following standard treatment.

ClinicalTrials.gov currently lists 55 study locations.³

The trial is described by Nuvation as global.

Yet, at the time of writing, the current ClinicalTrials.gov record does not list a UK centre.

That is not criticism of Nuvation Bio. Companies choose trial locations for numerous scientific, regulatory, financial and practical reasons.

But it is a fact that cannot be ignored.

It is also, for now, a moving target. Nuvation finalised the protocol amendment establishing SIGMA’s pivotal Phase 3 portion - enrolling around 300 patients across the United States, Australia and China - on 9 February 2026, and sites are still being added under that amendment as the trial recruits through to its estimated 2030 completion.⁴ If a UK arm is ever going to be attached to this trial, the window to make that case is open now, not once recruitment is complete.

Then there is G209

Perhaps even more interesting is Nuvation’s newly announced G209 Phase II study.

This will investigate safusidenib in people with Grade 2 or Grade 3 IDH1-mutant glioma whose disease has progressed after treatment with vorasidenib.¹

That is an enormously important question. Vorasidenib is now available to eligible UK NHS patients.⁸ Some of those patients may remain stable for years. But eventually we are going to have people in the UK asking:

‘Vorasidenib worked for me. Now it has stopped working. What comes next?’

Nuvation is about to investigate precisely that question - but G209 is currently planned for the United States only.

That seems to be exactly the sort of gap we ought to be identifying now, not several years from now when patients urgently need the answer.

The UK isn’t starting from scratch

We are not proposing that somebody suddenly construct a neuro-oncology clinical-trial system in Britain from nothing. The infrastructure already exists.

The Royal Marsden and The Christie, for example, are listed as UK centres in Servier’s clinical study combining vorasidenib with temozolomide in IDH-mutant glioma.⁵

UK centres therefore already have experience and infrastructure relevant to IDH-inhibitor trials.

We also understand that the Royal Marsden has indicated that it could provide infrastructure for a safusidenib study if such a trial were brought to the UK.

So the question is not simply:

‘Why can’t the UK run sophisticated molecular brain-tumour trials?’

We clearly can. The better question is:

‘How do we identify the trials UK patients most need - and then make a coordinated case for bringing them here?’

The letter-writing problem

At present, many of us in the brain-tumour community are working hard in the most traditional British fashion.

• We write to our MP.

• We ask the MP to write to the Health Secretary or relevant minister.

• We hope somebody in government contacts the appropriate department.

• We hope that department engages with the company.

• We hope the company responds.

• And then we wait.

It is worthwhile, of course. MPs should know that their constituents care about access to clinical trials, and ministers should be asked what they can do to make Britain attractive to companies such as Nuvation Bio.

But as a mechanism for identifying national clinical-research priorities, it is inevitably fragmented.

One family writes from London.

Another writes from Manchester.

Another campaign contacts a minister.

A charity makes representations elsewhere.

Everyone pushes the same boulder from a slightly different direction.

Progress can feel like wading through treacle.

ACT-BT could provide something much harder for the system to ignore… evidence.

And here’s the interesting bit: ACT-BT has already thought of this

The Brain Tumour Charity lists five things it hopes ACT-BT will achieve.

Number three is:

‘Build the case for more brain tumour clinical trials.’⁶

That is so important. The Brain Tumour Charity explicitly recognises that strict trial eligibility means some people referred to ACT-BT will have no suitable study available.

Rather than regarding those referrals as failures, ACT-BT intends to collect and collate that information.

The stated purpose is to identify groups within the brain-tumour community for whom no relevant research study exists and use that evidence to strengthen the case for investment in more trials.

In other words: ACT-BT may already be growing another limb. The questions are: how far can we take it and what should it look like?

What about an ACT-BT ‘Trial Gap Map’?

ACT-BT should not become a pressure group choosing individual drugs and declaring that they work before trials have proved it.

Nor should a panel, whose job it is to match patients to studies, be expected to negotiate commercial trial agreements with pharmaceutical companies.

But the information passing through ACT-BT could potentially become extremely valuable if aggregated and anonymised.

Imagine a regular, diarised ACT-BT Trial Gap Report answering four simple questions:

1. Which types and molecular subtypes of brain tumour are generating referrals for which no appropriate UK trial exists?

2. Which experimental treatments repeatedly appear relevant to UK patients but are available only through trials overseas?

3. Which UK neuro-oncology centres already possess the expertise and infrastructure that could potentially host those studies?

4. Where is the greatest measurable unmet need for a new clinical trial?

That would turn anecdote into evidence.

Instead of a patient group saying:

‘We really wish there was a safusidenib trial in the UK’,

we might eventually be able to say:

‘During the past year ACT-BT reviewed X patients with this molecular profile for whom there was no suitable UK study, while a relevant industry-sponsored programme was recruiting overseas.’

That is a completely different proposition.

It gives charities something concrete to campaign on, and it gives researchers something concrete to design trials around.

It also gives pharmaceutical companies evidence that a recruitable UK patient population exists, and it gives ministers something specific on which to act.

This fits the Government’s own policy

There is a further reason why now is the right time.

The Government’s Life Sciences Sector Plan says explicitly that Britain wants to become a much more attractive location for commercial clinical research. Its commitments include reducing commercial trial set-up times, increasing the number of people recruited to commercial interventional trials, expanding dedicated trial capacity and making NHS research performance more transparent.⁷

Government also says commercial research gives NHS patients access to cutting-edge treatments before those treatments become widely available.

So, there should be no philosophical disagreement here. The Government says:

We want more commercial clinical trials in Britain.

The brain-tumour community says:

We desperately need more clinical trials in Britain.

ACT-BT could increasingly tell both sides:

Here are the specific trials we are missing.

That begins to look less like campaigning and more like clinical-trial intelligence.

Safusidenib illustrates the opportunity

None of this means that Nuvation Bio should automatically bring every safusidenib study to the UK.

There are good reasons why particular trials are designed for particular countries. For example, Nuvation’s new G307 Phase III study in newly diagnosed Grade 2 glioma is specifically intended for regions outside the United States where vorasidenib is not yet approved or accessible.¹ Britain now has NHS access to vorasidenib, so G307 may simply not be the appropriate British study. Fair enough.

But that makes the broader question more important, not less.

What about SIGMA, the global high-risk study?

What about G209, investigating what happens after vorasidenib progression?

Could a British arm be added?

Could a related UK study be established?

What would Nuvation require from the NHS, NIHR, regulators and participating centres?

Is the obstacle cost?

Patient numbers?

Regulatory complexity?

Commercial priorities?

Trial set-up time?

Or has nobody yet put a sufficiently organised UK proposition in front of the company?

We don’t know - and that is precisely why somebody should ask.

From matching patients to trials - to matching trials to patients

ACT-BT is an excellent development because it reverses an old and unfair burden.

Instead of expecting an ill patient or frightened family to scour the world looking for a clinical trial, specialists now help identify the relevant research.

But perhaps ACT-BT can ultimately reverse another burden too.

Rather than waiting passively for pharmaceutical companies and academic sponsors to decide which studies arrive in the UK, its accumulated information could help to identify which studies UK patients actually need.

That would be a subtle but profound change.

At the moment the process largely runs one way:

Trial exists → find eligible patient.

Perhaps ACT-BT’s data could allow another route:

Patients exist → identify missing trial → demonstrate demand → approach sponsor.

ACT-BT gives us the map

On 30th August 2026 (yesterday) we described ACT-BT as a potentially important new piece of infrastructure for Britain’s brain-tumour community.

Having looked at it again, we think we may have underestimated it.

Its greatest contribution may eventually be not merely telling individual patients which roads are open to them.

It may be showing us where the roads haven’t yet been built.

Safusidenib is a useful example today. Tomorrow it may be a CAR-T therapy, a vaccine, focused ultrasound, an oncolytic virus or something none of us has yet heard of.

We cannot demand that every experimental treatment is trialled in Britain. We cannot assume that every promising early result will eventually produce a successful medicine. And government cannot order a private biotechnology company to open a British trial.

But we can become much better at identifying the gaps.

We can show companies where the patients are.

We can show ministers where the obstacles are.

We can show researchers where clinical need is accumulating.

And when a promising drug is being tested abroad, UK patients are potentially eligible for the science, and UK hospitals have the capability to conduct the work, we can at least ask a very simple question:

Why isn’t the trial here?

ACT-BT may be uniquely placed to help us provide the answer.

Links and further reading:

1. Nuvation Bio, “Nuvation Bio Announces Positive Updated Phase 2 Data and Expansion of Safusidenib Clinical Program with Two New Studies to Explore Broad Spectrum of IDH1-Mutant Glioma” (covers J201 data, and the new G209 and G307 studies), PR Newswire, 20 July 2026. Link

2. “Nuvation Bio Granted FDA Fast Track Designation for Safusidenib for Treatment of IDH1-Mutant Glioma,” PR Newswire, 20 August 2026. Link

3. SIGMA trial record (NCT05303519), including current listed study locations, ClinicalTrials.gov. Link

4. “Nuvation Bio Announces Pivotal Global Phase 3 SIGMA Trial (G203) for Safusidenib in IDH1-Mutant Glioma,” confirming the protocol amendment enrolling ~300 patients across the US, Australia and China, Nuvation Bio Investor Relations, 9 February 2026. Link

5. “Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma,” trial record listing Royal Marsden and Christie Hospital as UK sites (NCT06478212), ClinicalTrials.gov. Link

6. “Launching ACT-BT: Five ways we hope to make a difference,” The Brain Tumour Charity. Link

7. Life Sciences Sector Plan, HM Government (Department for Science, Innovation and Technology / Department for Business and Trade / Department of Health and Social Care), July 2025. Link

8. “Vorasidenib approved for NHS use in England,” The Brain Tumour Charity. Link

This week sees a spread of useful progress:

1. AI-assisted surgery

2. Glioblastoma recurrence biology

3. Radiotherapy research

4. AI learns to flag its own uncertainty

5. and 6. Two positive surveys on vorasidenib

But first, we lead with ACT-BT - not actually news this week, but it’s important that everyone should know what it is and how it should work.

ACT-BT means ‘Access to Clinical Trials for Brain Tumours’.

It is not a drug trial itself. It is a new UK-wide clinical-trial access pathway for brain tumour patients. In plain English: it is designed to stop patients and families having to trawl the internet themselves trying to work out which brain tumour trials might be suitable.

We know about the existence of trials like VIGOR, SIGMA, G209, ViCToRy, vorasidenib combinations, and vaccine studies, (see our special edition of 26^th August ‘The Push Toward Combination Therapy’) but ACT-BT takes us further by providing an infrastructure that may help patients actually find and gain access to these emerging treatments.

The ACT-BT project is funded and co-created by the Brain Tumour Charity.

What has happened so far:

The project was announced in January 2026 as a UK-wide initiative hosted by the University of Leeds, with Professor Susan Short as the ACT-BT lead. At that stage it was still being set up.

It then opened for referrals on 1 July 2026. Healthcare professionals can now refer people over 16 with a primary brain tumour from anywhere in the UK to a panel of clinical-trial experts.

So, to be very clear, this is not an NHS drug trial. It is a structured framework within which patients and families can navigate the complex clinical trials system and have their cases heard, not just by their individual oncology teams, but also by an organised body of professionals representing the brain cancer research community.

Who can be referred

The published patient information says any person over 16 diagnosed with a primary brain tumour can be referred, but only with their agreement. It does not matter whether they are newly diagnosed, recurrent, or already treated; however, individual trial eligibility will still depend on criteria specified by the team running the trial.

That is important. ACT-BT may identify trials, but it does not override trial inclusion criteria.

Who can make the referral

Patients cannot simply self-refer. Referral must come through:

• an oncologist,

• a Clinical Nurse Specialist with oncologist sign-off,

• or a research nurse with oncologist sign-off.

During the pilot phase, ACT-BT is not currently accepting referrals from other healthcare professionals, which may be a problem for some low-grade glioma patients who are not currently under an oncologist. The Brain Tumour Charity acknowledges this as an issue.

The process is as follows:

1. The patient agrees to be referred.

2. The clinician completes and emails the ACT-BT referral form.

3. The case is assigned a reference number and scheduled for panel review.

4. This panel of multi-disciplinary experts - each the lead researcher of a national study, so able to give accurate, current information on recruitment status - meets weekly to review cases and give individualised advice on which trials the patient may be eligible for.

5. This advice is sent back to the referring clinician within 24 hours.

6. The clinician discusses the options with the patient within about one week.

7. If a suitable trial is identified and the patient wants to proceed, the clinician then makes the actual trial referral.

During the pilot phase, they aim to review cases within about 10 days and provide feedback within two weeks of referral.

What information is needed

The referral requires quite a serious clinical dataset, including diagnosis, pathology, molecular pathology/genomic reports, treatment history, imaging reports, performance status, drug history, steroids, anti-epileptics, comorbidities, toxicities and any specific questions for the panel.

That tells us something useful: ACT-BT is not just a casual signposting service. It is trying to make proper trial-matching decisions from real clinical details.

Why it matters

The Brain Tumour Charity says brain tumour trials have the lowest recruitment levels of all cancer types in the UK, and its own barriers report found that only 12% of surveyed brain tumour patients had taken part in a clinical trial. Key barriers include lack of awareness, uneven geographical access, and insufficient support for patients’ cognitive and physical wellbeing.

This is exactly the gap ACT-BT is trying to fill: not inventing new treatments, but getting more patients into the trials that already exist.

What it is not

ACT-BT is not:

• a new therapy;

• a clinical trial itself;

• a guarantee of trial entry;

• a substitute for an oncology team;

• a way to bypass eligibility criteria;

• a patient self-referral portal;

• or a cure pathway dressed up as administration.

The final decision on eligibility remains with the team running the recruiting trial. Even if ACT-BT identifies a possible study, the patient may still be found ineligible at screening.

The strategic importance

ACT-BT is about infrastructure.

The brain tumour field is increasingly full of highly specific trials: IDH status, MGMT status, H3 K27M status, EGFR amplification, grade, recurrence status, previous treatment, steroid dose, performance status, surgical timing, location, scan characteristics, and more. That is too much for most families to navigate through.

ACT-BT could become a kind of national clearing house for brain tumour trial access.

Contributors:

Originator / Lead: Professor Susan Short, Professor of Clinical Oncology and Neuro-Oncology at the University of Leeds and co-director of the Leeds Cancer Research Centre, who put forward and leads the initiative (University of Leeds; BNOS).

Funder and co-creator: The Brain Tumour Charity, funding set-up and running costs - including a coordinator post and panel members’ time - for an initial eight-month pilot phase (The Brain Tumour Charity).

Patient involvement (PPIE): brainstrust, overseeing the Patient and Public Involvement and Engagement element; co-investigator Dr Helen Bulbeck, brainstrust co-founder (The Brain Tumour Charity).

Trial-matching partner: myTomorrows (The Brain Tumour Charity).

Host: University of Leeds / Leeds Cancer Research Centre (University of Leeds).

Reporting source: this item is compiled from The Brain Tumour Charity’s ACT-BT announcement and BNOS launch materials, the University of Leeds announcement, and the Let’s Talk About Brain Tumours podcast (Episode 68, April 2026, featuring Prof Susan Short) (podcast).

Okay, now for this week’s news round-up.

1. World-first: AI supports neurosurgeon in real time during live brain tumour surgery

Details:

• A UCL-developed AI system supported consultant neurosurgeon Professor Hani Marcus during surgery to remove a pituitary tumour from patient Rhys Hibbert at the National Hospital for Neurology and Neurosurgery, UCLH

• In this region of the brain, the pituitary gland, blood vessels and the nerves controlling vision sit tightly packed together; the AI helped highlight critical structures to avoid while removing as much tumour as safely possible

• The system was trained on hundreds of annotated endoscopic pituitary surgery videos and runs on an NVIDIA Clara IGX platform for real-time use in theatre

• Rhys’s sight, which had been under threat, was protected; within a week he was walking independently without glasses or a stick

Why it matters:

This is a genuine first - AI analysing the live surgical video feed itself, in real time, rather than pre-surgery scans, to help protect critical structures during one of the most delicate procedures in neurosurgery.

Caveat:

This is not an anti-cancer drug story, but it is very relevant to brain tumour treatment: better intraoperative navigation may help surgeons remove tumour more safely while reducing damage to critical structures. But this is surgical technology, not a cure.

Contributors:

Originator: Professor Hani Marcus and Danyal Khan, UCL Queen Square Institute of Neurology / UCL Hawkes Institute; technical lead Dr Sophia Bano, UCL Computer Science
Clinical Sponsor: National Hospital for Neurology and Neurosurgery, UCLH
Funder: National Institute for Health and Care Research (NIHR) and Google; supported by the NIHR Biomedical Research Centre at UCLH, Royal College of Surgeons, EPSRC and Wellcome
Campaigning Organisation: Not applicable
Reporting Source: UCL News, 26–27 August 2026

Link:

UCL news release

2. Drug repurposing targets the glioblastoma cells surgery leaves behind

Details:

• Researchers at the Brain Tumour Research Centre of Excellence, University of Nottingham, led by Dr Phoebe McCrorie, compared proteins on patient-derived glioblastoma cells against healthy brain cells

• They identified 18 membrane proteins consistently raised across multiple patient-derived cell lines, including LOXL1, A2M and SH3KBP1

• Computer-based screening flagged two already-approved drugs as worth investigating against these targets: nilotinib (leukaemia) and darifenacin (overactive bladder)

Why it matters:

No new glioblastoma treatment has been approved in over 20 years. This targets the ‘invasive edge’ cells that survive surgery and drive recurrence - and does it via drug repurposing, which could move faster than a novel compound.

Caveat:

This is laboratory cell-line work - the targets still need validating in patient samples, and there’s no indication yet these specific drugs would actually reach or work against tumour tissue in a real patient.

Contributors:

Originator: Dr Phoebe McCrorie, Brain Tumour Research Centre of Excellence, University of Nottingham
Clinical Sponsor: Not applicable - laboratory research
Funder: Brain Tumour Research
Campaigning Organisation: Brain Tumour Research
Reporting Source: Brain Tumour Research, 26 August 2026

Link:

Brain Tumour Research announcement

3. Australian researchers get first funding under new national brain cancer radiobiology initiative

Details:

• Professor Ivan Kempson, Adelaide University, has received A$240,000 to question decades-old assumptions about how radiotherapy affects tumour cells, healthy cells and the immune system - much of the current understanding dates to research from the 1960s-70s

• The work uses tools new to Australia that measure radiation-induced DNA damage with unprecedented precision, working directly with clinicians at Royal Adelaide Hospital using the same equipment used to treat patients

• This is the first of three planned ‘Innovation Partner Node’ projects under a new national coordination effort

Why it matters:

Radiotherapy is one of the pillars of brain tumour treatment, but it is still a blunt tool in many settings. This is a research-infrastructure/funding story aimed at making radiotherapy more precise and biologically informed.

Contributors:

Originator: Professor Ivan Kempson, Adelaide University Future Industries Institute
Clinical Sponsor: Royal Adelaide Hospital (clinical research setting)
Funder: Mark Hughes Foundation Centre for Brain Cancer Research, University of Newcastle (Australia)
Campaigning Organisation: Mark Hughes Foundation
Reporting Source: EurekAlert! / Adelaide University, 27 August 2026

Link:

EurekAlert! release

4. AI learns to flag its own uncertainty when measuring meningiomas on MRI – this work aims to make AI brain-tumour imaging more trustworthy

Details:

• UCSF researchers built an AI system that not only segments meningiomas on MRI in 3D but generates an ‘uncertainty map’ showing where its own measurement might be unreliable - aimed at the trust problem that limits clinical adoption of AI imaging tools

• Trained on 1,655 MRIs and externally validated in 353 patients

• The uncertainty maps aligned with the boundary areas radiologists themselves found ambiguous

Why it matters:

AI is only useful in clinical practice if clinicians know when to trust it and when not to. This is a diagnosis/planning story rather than a treatment story, but it fits your hierarchy because better measurement affects surgery, radiotherapy planning and follow-up decisions.

Caveat:

Multicentre and multi-rater validation is still needed before this progresses toward clinical use.

Contributors:

Originator: Andreas Rauschecker, MD, PhD, and colleagues, UCSF
Clinical Sponsor: Not applicable - computational/imaging research
Funder: Not stated in available reporting
Campaigning Organisation: Not applicable
Reporting Source: MedicalXpress, 24 August 2026; published in npj Digital Medicine

Link:

MedicalXpress coverage

And now, items 5 and 6 are about two surveys on vorasidenib – one patient and one clinical:

5. New patient survey adds weight to the case against NICE’s vorasidenib decision

Details:

• Following NICE’s draft decision not to recommend vorasidenib for NHS use, The Brain Tumour Charity surveyed the UK low-grade glioma community: 1,316 responses, 73% from patients or carers

• Support for NHS access was almost unanimous - 99% - and 86% identified a clear unmet clinical need

• Presented as an oral abstract at the BNOS 2026 / Neuro-Oncology supplement, 27 August 2026

Why it matters:

This shows how patient and clinician testimony can become part of the formal record around targeted-therapy availability.

Contributors:

Originator: Rachel Roberts and colleagues, The Brain Tumour Charity
Clinical Sponsor: Not applicable - patient survey
Funder: The Brain Tumour Charity
Campaigning Organisation: The Brain Tumour Charity
Reporting Source: Neuro-Oncology, Volume 28 Supplement 1, 27 August 2026

Link:

Abstract, Neuro-Oncology

6. First real-world data on how UK NHS trusts are using vorasidenib

Details:

• A companion abstract at the same BNOS 2026 proceedings surveyed 30 UK neuro-oncology centres: 20 of 30 are already using vorasidenib in adult patients despite NICE’s non-recommendation, via managed access arrangements

• No major safety concerns reported; barriers cited were clinic capacity, staffing, funding processes, and awaiting NICE guidance

• Regional disparities in access were noted between centres

Why it matters:

Evidence from real-world use will become increasingly important now that vorasidenib is moving from trial data into NHS practice.

Contributors:

Originator: Abraham Sondhi and Professor Susan Short, University of Leeds / British Neuro-Oncology Society
Clinical Sponsor: Not applicable - clinical survey
Funder: Not stated
Campaigning Organisation: British Neuro-Oncology Society (BNOS)
Reporting Source: Neuro-Oncology, Volume 28 Supplement 1, 27 August 2026

Link:

Abstract, Neuro-Oncology

And that’s it for this week.

What comes next for IDH inhibitors? Vorasidenib, safusidenib – and the push toward combination therapy

Inhibitors are changing the treatment landscape for IDH-mutant gliomas, and in doing so have created a new question: What comes next?

Researchers are now testing whether IDH inhibition can be pushed further – into higher-risk disease, after chemoradiotherapy, after resistance to another IDH inhibitor, and in combination with chemotherapy, checkpoint immunotherapy and cancer vaccines.

There is more than one IDH inhibitor in this story: alongside vorasidenib and the rapidly advancing safusidenib programme sits olutasidenib, an IDH1 inhibitor already approved for a blood cancer but also showing preliminary activity in heavily pre-treated glioma.

Much of what follows remains investigational, and trial eligibility, recruitment and access vary by country and can change quickly. But the trajectory is onward – and, for the most part, upward.

1. Vorasidenib – the drug that proved the principle

Vorasidenib is an oral, brain-penetrant inhibitor of mutant IDH1 and IDH2, and became the first IDH-directed drug approved specifically for glioma when the US FDA approved it in August 2024 for certain patients with Grade 2 IDH-mutant astrocytoma or oligodendroglioma following surgery.

Its significance isn’t just that it’s another anti-cancer drug: it changed a period of glioma treatment that previously meant largely watching and waiting – surveillance after surgery until progression made radiotherapy and chemotherapy necessary. Vorasidenib introduced another possibility: active treatment during that interval.

What the latest INDIGO data shows

Extended follow-up from the Phase III INDIGO trial, with a median follow-up of 41.6 months, was presented at ASCO 2026. Median progression-free survival by blinded independent review reached 44.1 months for patients receiving vorasidenib, up from 27.7 months at the original 2023 analysis. Only 23.8% had needed a further intervention – radiotherapy, chemotherapy or surgery – by that point, and the objective response rate had grown to 20.8%, continuing to deepen the longer patients stayed on treatment.

That matters for patients with lower-grade glioma, who can live for many years and for whom delaying radiotherapy and chemotherapy’s long-term effects matters enormously.

Why it matters

Vorasidenib established something fundamental: IDH mutation is not merely a diagnostic feature of glioma. It is therapeutically targetable. The drug does not cure glioma, and progression or resistance can eventually occur, nor do we yet know whether delaying progression will translate into longer overall survival. But the principle has been established.

UK and European access

The position has changed dramatically over the past year: NICE recommended vorasidenib for eligible patients in England in April 2026 (meaning it must be routinely funded by the NHS), NHS Scotland accepted the drug in March 2026, and Voranigo received EU-wide marketing authorisation in September 2025.

Contributors:

Originator: Agios Pharmaceuticals

Current developer / clinical sponsor: Servier

Funder: Industry-sponsored development programme

Key study: INDIGO – NCT04164901

Links:

INDIGO – ClinicalTrials.gov

Updated INDIGO results – ASCO 2026 (JCO)

NICE guidance – vorasidenib

Scottish Medicines Consortium decision

European Medicines Agency – Voranigo

2. Safusidenib – expanding across the IDH1-mutant disease pathway

Safusidenib is an oral, brain-penetrant selective mutant-IDH1 inhibitor that began life as DS-1001/DS-1001b at Daiichi Sankyo; Nuvation Bio subsequently acquired the programme and now holds global development and commercialisation rights.

Safusidenib has moved quickly in 2026: on 20 August the US FDA granted it Fast Track Designation for IDH1-mutant glioma – not approval, but a status that allows closer FDA interaction and can accelerate development and review if trial results stay positive.

The encouraging Grade 2 data

Updated results from the Japanese Phase II J201 study – 27 patients with Grade 2 IDH1-mutant glioma who had not previously received chemotherapy or radiotherapy – showed, at a median follow-up of 38.8 months, a confirmed objective response rate of 51.9%, a median progression-free survival not yet reached, and 79.1% progression-free at three years, with no new safety signal. Encouraging, but from a small, non-randomised study needing confirmation in larger trials.

But safusidenib is no longer just a Grade 2 story

Nuvation is now testing safusidenib across several stages of IDH1-mutant glioma. The SIGMA Phase III programme (currently recruiting) pairs maintenance safusidenib against placebo after standard radiotherapy or chemoradiotherapy and temozolomide, in higher-risk Grade 2/3 astrocytoma and Grade 4 IDH1-mutant astrocytoma, plus a cohort in residual or recurrent Grade 3 oligodendroglioma following surgery.

Nuvation has also launched two further studies: G307 (Phase III) – safusidenib in newly diagnosed Grade 2 IDH1-mutant glioma outside the United States; and G209 (Phase II) – safusidenib in patients whose IDH1-mutant glioma has progressed after treatment with vorasidenib. That second study could become particularly important: for the first time, researchers are asking not simply whether an IDH inhibitor works, but what happens when the first one stops working.

Why it matters

Vorasidenib established IDH inhibition in relatively early Grade 2 disease; safusidenib is now asking whether it can work after chemoradiotherapy, in higher-risk disease, or after vorasidenib progression – less like one successful drug than the emergence of an IDH-treatment strategy.

Contributors:

Originator: Daiichi Sankyo – originally DS-1001/DS-1001b

Current developer / clinical sponsor: Nuvation Bio

Funder: Nuvation Bio / industry-sponsored clinical programme

Key study: SIGMA – NCT05303519

Links:

Nuvation Bio – FDA Fast Track announcement

Nuvation Bio – July 2026 programme expansion

SIGMA – ClinicalTrials.gov

G209 – post-vorasidenib safusidenib study

G307 – Grade 2 safusidenib Phase III

3. VIGOR – vorasidenib after chemoradiotherapy?

One of the most important developments since INDIGO is the VIGOR trial (EORTC-2427-BTG). Where INDIGO asked whether vorasidenib could delay progression in patients who’d had surgery but didn’t yet need radiotherapy or chemotherapy, VIGOR asks something different: could vorasidenib help after chemoradiotherapy has already been given?

It is a randomised, placebo-controlled, triple-blind Phase III study of vorasidenib as maintenance treatment following first-line chemoradiotherapy in patients with IDH-mutant Grade 2 or 3 astrocytoma, aiming to enrol approximately 468 patients; the first patient was randomised in January 2026, and recruitment is now international.

UK centres listed by EORTC for activation include The Royal Marsden (Sutton), The Christie (Manchester), Clatterbridge Cancer Centre, and Queen Elizabeth Hospital Birmingham – though this can change, so check the current trial record before assuming a centre is open.

Why it matters

If VIGOR succeeds, the role of vorasidenib could extend well beyond the relatively favourable population studied in INDIGO – moving IDH inhibition towards becoming a maintenance treatment after conventional therapy, rather than an alternative used only before it.

Contributors:

Originator / coordinator: EORTC Brain Tumour Group

Clinical collaborators: Servier, Canadian Cancer Trials Group and Cooperative Trials Group for Neuro-Oncology

Study coordinators: Professor Matthias Preusser and Dr Marjolein Geurts

Trial: NCT06809322

Links:

EORTC VIGOR study page

VIGOR first-patient announcement

VIGOR – ClinicalTrials.gov

4. The next frontier – combining IDH inhibitors with other treatments

This may become the biggest story of all: IDH inhibitors work against an important driver of IDH-mutant glioma but don’t eliminate every tumour cell, so researchers are asking whether IDH inhibition can be the foundation on which another treatment is added. Three approaches are already being tested.

Vorasidenib + temozolomide

A Phase Ib/II study of vorasidenib plus the chemotherapy drug temozolomide enrolled 51 participants and is active but no longer recruiting; its Phase II component focuses on Grade 4 IDH-mutant astrocytoma following radiotherapy and concurrent temozolomide, at UK centres including The Christie (Manchester) and The Royal Marsden (Sutton).

The question: could continuing IDH suppression make temozolomide more effective, or prolong the benefit achieved by conventional treatment?

Trial: Vorasidenib + temozolomide – NCT06478212

Vorasidenib + pembrolizumab

Pembrolizumab is a checkpoint inhibitor, designed to release one of the biological brakes that prevents T cells attacking cancer. Checkpoint inhibitors have transformed treatment in several cancers but have largely disappointed when used alone in glioma, possibly because the tumour is too immunologically suppressive for checkpoint blockade alone.

A Phase I study of vorasidenib plus pembrolizumab in recurrent or progressive IDH1-mutant glioma enrolled 60 patients and is active but no longer recruiting.

The biological logic: mutant IDH produces the oncometabolite D-2-hydroxyglutarate (D-2-HG), which, beyond altering tumour metabolism, contributes to an immunosuppressive tumour environment. IDH inhibition reduces D-2-HG, so the theory is to first weaken the tumour’s immune defences with an IDH inhibitor, then let checkpoint immunotherapy attack. Whether that translates into meaningful benefit remains unproven.

Trial: Vorasidenib + pembrolizumab – NCT05484622

Vorasidenib + an IDH1 tumour vaccine

The ViCToRy trial at Duke University takes this a step further (see Item 2(d)(ii) of the 25.2.26 BTNL edition), combining vorasidenib with a peptide vaccine known as PEPIDH1M, designed to provoke an immune response against the IDH1-mutant tumour. The Phase I study is currently recruiting at Duke, building on earlier work showing that vaccines directed at the characteristic IDH1-R132H mutation can provoke measurable tumour-specific immune responses – including the German studies NOA-16 (NCT02454634) and AMPLIFY-NEOVAC (NCT03893903), which combined an IDH1 vaccine with the PD-L1 checkpoint inhibitor avelumab.

Why it matters: the same immune-priming logic applies here as with pembrolizumab – a compelling hypothesis, but not yet a proven treatment strategy.

Trials:

ViCToRy – NCT05609994

NOA-16 – NCT02454634

AMPLIFY-NEOVAC – NCT03893903

5. What are these combination trials trying to solve?

The emerging strategy reduces to four problems:

1. Residual tumour survives – an IDH inhibitor slows the tumour without eliminating every malignant cell. Possible answer: combine with chemotherapy.

2. Glioma suppresses immune attack – IDH-mutant tumours make immune cells less effective. Possible answer: inhibit mutant IDH, then add checkpoint immunotherapy.

3. The immune system doesn’t recognise the tumour. Possible answer: combine an IDH inhibitor with a vaccine directed at the mutant IDH protein.

4. Resistance eventually develops – a patient may progress despite one IDH inhibitor. Possible answer: determine whether another IDH inhibitor still works, or whether resistance needs a different strategy altogether – which is why Nuvation’s post-vorasidenib G209 safusidenib study could be particularly informative.

The caveats

In a field moving this quickly, there’s a danger that a run of promising trials starts to look like a run of proven treatments. It isn’t.

Gliomas remain immunologically difficult: checkpoint inhibitors alone have largely failed to reproduce the successes seen in melanoma or lung cancer. Combining them with IDH inhibition is scientifically rational, but still experimental.

Enhancing disease remains difficult: once an IDH-mutant tumour becomes contrast-enhancing and biologically more aggressive, response to IDH inhibition has generally been much less impressive. Safusidenib and olutasidenib show signals of activity here, but the evidence remains far too limited to claim either has solved the problem.

Resistance is becoming the next question. As more patients stay on IDH inhibitors for longer, we’ll learn more about how tumours adapt. The field has to move beyond ‘Which IDH inhibitor works?’ towards ‘Which IDH inhibitor, at which stage, in which sequence, and combined with what?’

And throughout: a ‘Recruiting’ status on ClinicalTrials.gov doesn’t guarantee a given site is open. Always check the current trial record and eligibility criteria, and discuss participation with your own neuro-oncology team.

The bigger picture

Vorasidenib proved that IDH-mutant glioma can be attacked at a defining molecular vulnerability; safusidenib is testing how far that idea can be pushed; VIGOR is asking whether it should continue after chemoradiotherapy; other studies are combining it with chemotherapy, checkpoint inhibitors and vaccines; and olutasidenib shows there may be other IDH inhibitors, with different properties and niches, still to come.

So, the next stage in the IDH story may not be about finding the one best inhibitor – it may be about learning to use an expanding family of IDH-targeted treatments intelligently.

The bottom line

IDH inhibitors appear to be evolving from individual drugs into being part of a comprehensive, strategic treatment platform.

1. Safusidenib fast tracked

2. Laser surgery in difficult brain tumours

3. Brain-targeted T-cell engager

4. AI-designed “off-the-shelf” glioblastoma vaccine

5. Immune “command posts” inside skull bone marrow

6. Inhibitor drug attacks temozolomide-resistant glioma stem cells

7. Biomimetic nanoparticles cross the BBB

8. and 9. Positron Emission Tomography - what it is and how it could help

1. As we reported in our News Extra on 21^st August, the FDA has granted Fast Track status to safusidenib for IDH1-mutant glioma

The US Food and Drug Administration has granted Fast Track Designation to safusidenib, Nuvation Bio’s oral, brain-penetrant inhibitor of mutant IDH1, for the treatment of IDH1-mutant glioma.

Fast Track does not mean safusidenib has been approved. What it does mean is that the FDA considers the drug sufficiently promising, in an area of serious unmet medical need, to permit more frequent regulatory discussions and potentially a faster review process if the clinical evidence ultimately supports approval.

The decision comes as Nuvation’s clinical programme expands substantially. Its pivotal Phase III SIGMA study is already enrolling, while the company is also planning a Phase III G307 study outside the US and the especially interesting G209 Phase II trial in patients whose glioma has progressed after vorasidenib.

The FDA decision was supported by the existing clinical programme, including the Japanese Phase II J201 study. Those are not new results this week, but for context, Nuvation previously reported a 51.9% confirmed objective response rate in 27 chemotherapy- and radiotherapy-naïve Grade 2 IDH1-mutant patients, with 79.1% remaining progression-free at three years.

Caveat

Fast Track status is regulatory acceleration, not proof of efficacy and not marketing approval. Safusidenib still has to succeed in larger clinical trials.

Nevertheless, in terms of the drug’s journey towards becoming a possible second major IDH inhibitor after vorasidenib, this is a significant milestone.

Originator: Originally developed as DS-1001b by Daiichi Sankyo; current global safusidenib programme developed by Nuvation Bio

Clinical Sponsor: Nuvation Bio - current Phase III/expanding clinical programme

Funder: Nuvation Bio

Campaigning Organisation: None identified

Reporting Source: Nuvation Bio; independently reported by CancerNetwork and OncLive

Links: Nuvation Bio - FDA Fast Track announcement · CancerNetwork coverage · OncLive coverage

2. Large prospective study strengthens case for laser surgery in difficult brain tumours

17 August 2026 - United States

A major analysis from the LAANTERN registry has reported outcomes from 787 patients treated at 25 US centres with MRI-guided Laser Interstitial Thermal Therapy - LITT.

Rather than opening the skull to remove a tumour conventionally, surgeons insert a narrow laser probe through a small opening and heat tumour tissue while monitoring the procedure with MRI. This makes LITT particularly attractive for deep, difficult-to-access or recurrent tumours where conventional surgery may carry considerable risk.

The prospective multicentre study found that achieving a high degree of tumour ablation was strongly associated with better outcomes. The WashU-led investigators also reported relatively short hospital stays and preservation of quality of life, supporting the idea that LITT can provide a meaningful minimally invasive option for selected patients.

Why it matters

This is considerably more convincing than another small single-centre LITT case series. Nearly 800 prospectively followed patients across numerous centres provides a substantial real-world evidence base.

It also supports something we’ve increasingly seen across brain-tumour research: “surgery” is no longer necessarily synonymous with a large craniotomy.

Caveat

LAANTERN is a prospective registry, not a randomised trial comparing LITT directly with conventional surgery. Patient selection can therefore influence outcomes.

There is also an important commercial connection: the registry was sponsored and funded by Monteris Medical, manufacturer of the NeuroBlate laser system. The investigators retained responsibility for clinical interpretation and publication.

Originator: Professor Eric Leuthardt, Washington University School of Medicine, and the multicentre LAANTERN investigators

Clinical Sponsor: Monteris Medical Corporation - LAANTERN registry, NCT02392078

Funder: Monteris Medical Corporation

Campaigning Organisation: None identified

Reporting Source: Journal of Clinical Oncology / American Society of Clinical Oncology; Washington University reporting

Links: Peer-reviewed Journal of Clinical Oncology paper · ClinicalTrials.gov - NCT02392078

3. First-in-human trial opens for APTN-101 - a brain-targeted T-cell engager

20 August 2026 - Duke University, United States
This one is particularly interesting because it attempts to solve two problems at once: getting an immunotherapy into the brain and then directing the patient’s T cells specifically against tumour cells.

Adaptin Bio has opened enrolment in the first Phase I human trial of APTN-101, based on Duke University’s BRiTE - Brain Bispecific T-cell Engager - platform.

APTN-101 targets EGFRvIII, a tumour-associated mutation found in a subset of glioblastomas. It is designed both to cross the blood-brain barrier more effectively and to bring T cells into contact with EGFRvIII-positive glioma cells.

The small, open-label dose-escalation study at Duke will primarily establish safety and maximum tolerated dose, while also looking for early signals of tumour response, progression-free survival and overall survival. Adaptin says it expects to enrol up to 15 adults.

Preclinically, the company reports more than a seven-fold improvement in brain distribution compared with the T-cell engager alone and complete eradication of EGFRvIII-positive tumours in 70–80% of treated mice. Those figures are impressive, but they remain company-reported mouse results.

Why it matters

Bispecific T-cell engagers have become an important immunotherapy class in other cancers, but the BBB and glioblastoma’s immunosuppressive environment make brain tumours much harder.

APTN-101 is explicitly engineered around those difficulties rather than simply importing an existing cancer immunotherapy into GBM.

Caveat

No human efficacy data exist yet. This Phase I trial is primarily asking whether the treatment can be administered safely.

Originator: BRiTE platform developed by Duke University Department of Neurosurgery researchers; preclinical programme led by Professor Mustafa Khasraw and colleagues; APTN-101 developed commercially by Adaptin Bio

Clinical Sponsor: Investigator-led programme at Duke University; Professor Mustafa Khasraw is Principal Investigator, in collaboration with Adaptin Bio

Funder: Adaptin Bio/Duke development collaboration; Adaptin has sponsored research agreements with Duke

Campaigning Organisation: None identified

Reporting Source: Adaptin Bio/GlobeNewswire; Duke programme

Links: APTN-101 Phase I enrolment announcement · Independent report

4. AI-designed “off-the-shelf” glioblastoma vaccine EVX-05 enters development

17 August 2026 - Denmark/United States

Evaxion has announced a new therapeutic glioblastoma vaccine programme called EVX-05, being developed with experts at Duke University.

The idea is unusual. Many experimental cancer vaccines are personalised for an individual patient’s tumour. EVX-05 is intended instead to be off-the-shelf, targeting antigens shared across different glioblastomas.

Evaxion’s AI-Immunology platform has identified targets derived from endogenous retroviruses - ERVs: remnants of ancient viral DNA embedded in the human genome which can become abnormally activated in cancer cells. If the same immunogenic ERV-derived targets occur across many GBMs, researchers may be able to build a vaccine usable in more than one patient without manufacturing a bespoke product every time.

Evaxion says it is now optimising the lead vaccine candidate, with Professor Mustafa Khasraw’s Duke team expected to undertake initial clinical testing.

Why it matters

This combines three rapidly developing areas: cancer vaccination, tumour genomics and AI-assisted target discovery.

An effective off-the-shelf vaccine would also have obvious practical advantages over highly individualised vaccine manufacture.

Caveat

EVX-05 is not yet in patients. It is a newly announced development programme moving towards a possible Phase I study.

Originator: Evaxion A/S in collaboration with Duke University; Evaxion AI-Immunology platform

Clinical Sponsor: Not yet applicable - no clinical EVX-05 trial has begun

Funder: Evaxion A/S within its existing R&D programme

Campaigning Organisation: None identified

Reporting Source: Evaxion; independent biotechnology coverage from Fierce Biotech

Links: Evaxion EVX-05 announcement · Fierce Biotech coverage

5. Scientists discover immune “command posts” inside skull bone marrow - and exploit them against brain tumours in mice

19 August 2026 - Washington University, St Louis

This may be the most intriguing basic-science story of the week.

Researchers led by Jang Hyun Park and Professor Jonathan Kipnis have identified organised immune structures within the skull bone marrow that appear to act as local surveillance centres for the brain.

The work, published in Nature, suggests that rather than depending entirely on distant lymph nodes, the brain can call upon immune-cell populations housed immediately next door in the skull.

In mouse brain-tumour models, interfering with these structures accelerated tumour growth and shortened survival. Conversely, delivering immune-stimulating molecules in a gel beneath the scalp enhanced local immune responses and improved tumour rejection and survival. The researchers also identified corresponding immune-cell populations in human skull marrow, although their anti-cancer role in humans remains to be demonstrated.

Why it matters

There is a potentially important drug-delivery implication here.

Instead of trying to drive an immunotherapy through the blood-brain barrier, it may eventually be possible to stimulate immune activity in the skull immediately outside the brain and exploit the natural channels connecting skull marrow with the CNS.

That is a substantially different way of thinking about brain immunotherapy.

Caveat

The therapeutic experiment is still predominantly mouse research. Calling the structures a newly discovered “immune organ”, as some reports have done, is useful shorthand rather than a declaration that medicine has suddenly added a new anatomical organ to the textbooks.

Originator: Jang Hyun Park, Jonathan Kipnis and colleagues, Washington University School of Medicine

Clinical Sponsor: Not applicable - preclinical research

Funder: US National Institutes of Health; Cure Alzheimer’s Fund; BJC Investigators Program; National Research Foundation of Korea/Ministry of Education; ChadTough Defeat DIPG Foundation fellowship

Campaigning Organisation: ChadTough Defeat DIPG Foundation - also a research funder

Reporting Source: Nature; Washington University School of Medicine

Links: Nature research paper · WashU explanation

6. Abexinostat attacks temozolomide-resistant glioma stem cells

19 August 2026 - University of Nebraska Medical Center
Researchers have reported encouraging preclinical results with abexinostat, a histone deacetylase inhibitor, against one of glioblastoma’s most troublesome populations: glioma stem cells that persist despite temozolomide treatment.

Using patient-derived glioblastoma cultures and patient-derived mouse xenografts, the team found that abexinostat reduced several DNA-repair proteins, including RAD51, CHK1, Ku70 and MGMT, while also reducing markers associated with cancer-cell “stemness”.

It also reduced the ability of the stem-like cells to renew themselves and slowed tumour growth in mouse models. The researchers tested abexinostat in combination with temozolomide, raising the possibility that the drug might make the hard-to-kill reservoir of GBM cells more vulnerable to existing treatment.

Why it matters

One theory of glioblastoma recurrence is that ordinary treatment kills much of the rapidly dividing tumour while a highly resistant stem-like population survives, eventually rebuilding it.

If abexinostat can attack both DNA repair and the stem-cell phenotype, it could potentially target precisely that recurrence mechanism.

Caveat

This is cell and mouse-model research, not evidence of benefit in GBM patients.

Originator: Balaji Perumalsamy, Nicole Shonka, Surinder K. Batra and colleagues, University of Nebraska Medical Center/Fred & Pamela Buffett Cancer Center

Clinical Sponsor: Not applicable - preclinical research

Funder: Specific grant support could not be reliably established from the accessible accepted-manuscript metadata

Campaigning Organisation: None identified

Reporting Source: Neuro-Oncology, Society for Neuro-Oncology/Oxford University Press

Link: Original Neuro-Oncology paper

7. Biomimetic nanoparticles cross the BBB while combining chemotherapy and immune-checkpoint targeting

18 August 2026 - China

Researchers in Chengdu have engineered a remarkably complex nanoparticle designed to tackle several glioma problems simultaneously.

The nanoparticles contain elemene and cabazitaxel and are coated with membranes derived from mesenchymal stem cells engineered to overexpress PD-1.

The stem-cell membrane is intended to exploit the cells’ natural tumour-homing and BBB-crossing characteristics, while PD-1 allows the particles to interact with PD-L1 in the tumour environment.

The researchers report a 6.26-fold improvement in BBB crossing, an 8.57-fold improvement in tumour accumulation and, in an orthotopic mouse glioma model, a 57.5% extension in survival, along with increased anti-tumour immune activity and no obvious systemic toxicity.

Why it matters

This illustrates how sophisticated modern drug-delivery research is becoming. The nanoparticle is not merely a microscopic container for chemotherapy: its surface is engineered to cross the BBB, recognise the tumour and alter the immune environment simultaneously.

Caveat

It is also extremely complicated - and complicated laboratory technologies can become difficult to manufacture reproducibly and safely at clinical scale.

These are mouse and laboratory findings, not patient results.

Originator: Jie Li, Yuhao Gao, Wenbo Zhao and colleagues, Chengdu University of Traditional Chinese Medicine and Chengdu Fifth People’s Hospital

Clinical Sponsor: Not applicable

Funder: National Natural Science Foundation of China; China National Postdoctoral Program for Innovative Talents; China Postdoctoral Science Foundation; Natural Science Foundation of Sichuan Province; Chengdu Municipal Science and Technology Bureau; Xinglin Scholars programme

Campaigning Organisation: None identified

Reporting Source: Frontiers in Immunology

Link: Original Frontiers in Immunology paper

Imaging and monitoring: an unusually busy week

Your added Diagnosis / Monitoring headings were particularly useful here. Three developments would probably have escaped a normal treatment-focused search.

Explanatory note before you read the next two items:

PET stands for Positron Emission Tomography — an imaging technique that detects a radioactive tracer injected into the body, showing where it accumulates based on metabolic or biological activity, rather than just anatomical structure the way a standard MRI or CT does.

In the next two items in this week’s edition, the letters in front of “PET” tell you which tracer was used:

• PSMA PET uses a tracer that binds to prostate-specific membrane antigen — a protein that, helpfully for glioma imaging, also shows up on the blood vessels feeding high-grade brain tumours.

• FET PET uses a radiolabelled amino acid (fluoroethyltyrosine) that active, dividing tumour cells take up more than normal brain tissue does.

That’s the appeal of PET in neuro-oncology generally: standard MRI mostly shows where the blood-brain barrier has broken down (which can mean tumour, but can also mean inflammation, radiation effects, or scar tissue), whereas PET shows where cells are metabolically active - which is often a more direct read on whether it’s genuinely tumour.

Okay, now read on:-

8. PSMA PET may show glioblastoma beyond the margins visible on MRI

17 August, United States
A prospective study of 27 people with high-grade glioma tested PSMA PET both for radiotherapy planning and for distinguishing recurrence from post-treatment effects.

In four of the 19 patients being assessed for radiotherapy, the PSMA-positive tumour volume extended beyond the contrast-enhancing abnormality seen on MRI. Among eight patients investigated for suspected recurrence, PSMA PET correctly identified pathology-confirmed progression or necrosis in seven - 88%.

Why it matters

The implication is potentially important: PET might eventually help radiotherapists see biologically active tumour that conventional contrast MRI misses.

Originator: Rituraj Upadhyay and Pavnesh Kumar, co-first authors; Joshua Palmer and collaborators at Moffitt Cancer Center, Ohio State and partner institutions

Clinical Sponsor: Not applicable - prospective imaging registry

Funder: No specific study funding identified in the accessible publication record

Campaigning Organisation: None identified

Reporting Source: Neuro-Oncology Advances

Link: PSMA PET study

9. FET PET may reveal early whether recurrent GBM is responding to bevacizumab

17 August - Denmark
Researchers at Copenhagen’s Rigshospitalet examined 82 patients with recurrent IDH-wildtype glioblastoma who had received bevacizumab plus irinotecan and undergone FET-PET imaging before treatment and after two cycles.

Changes in PET metabolic tumour volume and tumour-to-background uptake predicted survival. In particular, reductions of more than 76% in metabolic tumour volume and more than 21% in maximum tumour-to-background signal were associated with longer survival.

Why it matters

Bevacizumab can make an MRI look better partly by reducing vascular leakage, meaning that apparent radiological improvement does not always equal tumour control.

FET PET measures tumour metabolism rather than simply contrast leakage and may therefore provide a better early indication of who is genuinely benefiting.

Originator: Shivani Chiranth and colleagues, Rigshospitalet/Danish Comprehensive Cancer Center Brain Tumor Center

Clinical Sponsor: Not applicable - retrospective clinical imaging cohort

Funder: Specific funding not identified in accessible publication metadata

Campaigning Organisation: None identified

Reporting Source: Neuro-Oncology Advances

Link: FET PET / bevacizumab paper

A busy week, and a lot to think about. As always, let me know how you think this substack could be improved.

On 20th August, 2026, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to safusidenib for the treatment of IDH1-mutant glioma.

FDA Fast Track Designation is intended to facilitate the development and expedite the regulatory review of drugs that treat serious conditions and address unmet medical needs. This designation can lead to more frequent interactions with the FDA and potentially faster approval if subsequent clinical trials (such as the ongoing Phase 3 SIGMA study) continue to show positive results.

Given the strong response-rate data behind the decision, this is a meaningful step for safusidenib.

Links:

Nuvation Bio Granted FDA Fast Track Designation for Safusidenib for Treatment of IDH1-Mutant Glioma - BioSpace

Nuvation Bio Granted FDA Fast Track Designation for Safusidenib for Treatment of IDH1-Mutant Glioma

A quieter week for brain tumour research, but not an empty one.

• Two very different drug-delivery approaches moved forward - one straight up the nose and the other implanted directly into the surgical space.

• Also, an intriguing electrical-field treatment produced its first results in a living brain.

1. Intranasal NEO100 meets primary endpoint in recurrent IDH1-mutant high-grade glioma

Why this is potentially good news

This targets a population that current IDH-targeted drugs don’t reach. Vorasidenib and safusidenib - the IDH inhibitors we’ve tracked closely - are aimed principally at newly diagnosed, lower-grade disease before radiotherapy and chemotherapy. NEO100-01 is being tested at the opposite end of the spectrum: Grade III/IV IDH1-mutant tumours that have already recurred after radiotherapy and temozolomide, where treatment options are limited.

Its method of delivery is also unusual. Rather than relying on a drug circulating in the bloodstream and crossing the blood-brain barrier, patients self-administer NEO100 as a nasal mist at home, with the intention of reaching the brain through the olfactory and trigeminal pathways - largely sidestepping the conventional blood-brain barrier problem.

The Phase 2a stage of the NEO100-01 trial treated 24 patients whose Grade III/IV IDH1-mutant glioma had already returned after radiotherapy and chemotherapy.

The trial met its primary endpoint: 48.9% of patients had no tumour progression at six months, more than double the trial’s pre-specified historical benchmark of 20%.

Two of the 24 patients experienced measurable tumour shrinkage, while five remained on treatment at the time of reporting.

One patient had remained progression-free for around 19 months, while another had maintained tumour shrinkage for more than 114 days.

Side effects were mostly mild and consistent with earlier safety testing; no major toxicities were reported.

NeOnc now intends to seek a Type B meeting with the US Food and Drug Administration to discuss a possible registrational development pathway.

Caveat

This was a small, single-arm study involving only 24 patients, with no control group being treated alongside them. Everyone received NEO100, while the 20% comparison figure came from historical data rather than contemporaneous patients.

That makes the result encouraging, but it does not carry the evidential weight of a randomised controlled trial.

Originator: NeOnc Technologies Holdings, Inc.; underlying perillyl-alcohol technology developed by Dr Thomas C. Chen and exclusively patent-licensed from the University of Southern California

Clinical Sponsor: NeOnc Technologies Holdings, Inc. - trial NCT02704858, conducted at multiple sites including Saint John’s Cancer Institute

Funder: NeOnc Technologies Holdings, Inc. - company-funded; no charity or government co-funder identified for this trial

Campaigning Organisation: Not applicable

Reporting Source: NeOnc Technologies press release via GlobeNewswire, 12 August 2026

Links: Topline results announcement · ClinicalTrials.gov - NCT02704858

2. Irinotecan-ChemoSeed clears a key hurdle ahead of Phase 2 glioblastoma recruitment

Why this matters

This sits in the same broad local drug-delivery category as chemotherapy wafers and brachytherapy implants such as GammaTile: putting treatment directly into the surgical margin, where glioblastoma most commonly recurs, rather than relying on a drug to reach the tumour from the bloodstream.

It is also particularly relevant to a question we have been examining recently in BTNL: could brain-tumour surgery become not only a means of removing disease, but an opportunity to deliver treatment directly to the place where recurrence begins?

This week’s news is not an efficacy result. It is a genuine operational milestone showing that the trial is moving closer to treating patients.

CRISM Therapeutics has completed the Site Initiation Visit at its lead NHS trial centre for the Phase 2 OPTICAL study, NCT07356973.

This removes an important operational hurdle before patient recruitment begins; recruitment is expected to start ‘in the coming weeks’.

ChemoSeed is an implantable, biodegradable polymer device placed directly into the tumour resection margin during surgery.

In this indication it is designed to release irinotecan locally and gradually over time.

The platform can, in principle, carry as many as four drugs in separate layers, each with different release characteristics.

The trial has two stages. The first will assess safety in patients whose glioblastoma has already recurred after surgery, beginning with a low dose of irinotecan and increasing it cautiously.

A larger stage is then planned in newly diagnosed patients, comparing ChemoSeed plus standard of care with standard of care alone.

The principal outcome researchers will follow is progression-free survival, with other measures including tumour response and longer-term disease control.

The programme already holds an MHRA Innovation Passport, awarded in August 2024, and FDA Orphan Drug Designation covering high-grade gliomas.

Caveat

This is a trial moving forward, not an available new treatment (yet). No efficacy signal has yet been reported from this study.

CRISM is also a small, early-stage biotechnology company, something worth bearing in mind when interpreting company announcements alongside the underlying science.

Originator: CRISM Therapeutics Corporation; Professor Chris McConville, Chief Scientific Officer at CRISM and Professor of Biomedical Innovation at Ulster University

Clinical Sponsor: CRISM Therapeutics Corporation

Funder: CRISM Therapeutics Corporation - company-funded; no charity or government grant identified specifically for this glioblastoma programme

Campaigning Organisation: Not applicable

Reporting Source: CRISM Therapeutics / market announcement, 13 August 2026

Links: Clinical-trial update · ClinicalTrials.gov - NCT07356973 · UK HRA study summary

3. Implanted ‘rotating’ electric field shrinks glioblastoma tumours in rat model

Why this matters

This is a genuinely different approach from the Tumour Treating Fields - TTFields - technology used clinically in glioblastoma.

TTFields are delivered through arrays placed externally on the scalp. The new approach, known as Intratumoral Modulation Therapy, or IMT, instead places electrodes directly around the tumour.

In principle, that means the electric field can be delivered more strongly and more precisely to the tumour itself.

It is still animal research and years from being tested in people, but it marks a genuine technical milestone in a programme that has been developing since 2018.

Published in Neuro-Oncology Advances, the study tested three electrodes implanted around a glioblastoma tumour.

The electrical signal was phase-shifted between the electrodes to create a dynamic or rotating electric field.

The aim is to expose the tumour more evenly and avoid the “cold spots” that a static field can leave.

This is the first time the researchers have achieved such a dynamic, multi-electrode field in a living brain; earlier work used cell cultures and simpler electrode arrangements.

After seven days of treatment in rats, researchers reported an eight-fold reduction in tumour growth measured by bioluminescence and a five-fold reduction in tumour volume measured by MRI, compared with untreated controls.

No neurological adverse effects or imaging evidence of brain injury were observed.

The same research group has previously reported effects against diffuse intrinsic pontine glioma - DIPG - cells, suggesting that the approach may eventually have applications beyond adult glioblastoma.

The team is now working on a prototype device.

Lead author Erin Iredale has suggested that an initial human clinical trial could still be five to ten years away.

Caveat

These are striking results, but they remain animal-model results.

A treatment that shrinks a tumour in a rat brain may fail for many reasons when translated into humans. The importance of this study is therefore not that a new therapy is about to become available, but that a technically difficult method of delivering a complex electrical field inside the living brain appears to have worked.

Originator: Dr Matthew Hebb and Erin Iredale, Western University’s Schulich School of Medicine & Dentistry, with Eugene Wong, Terry Peters and Susanne Schmid

Clinical Sponsor: Not applicable - preclinical animal-model research

Funder: Not stated in the reporting available for this edition

Campaigning Organisation: Not applicable

Reporting Source: Western University News, 13–14 August 2026, with subsequent coverage including MedicalXpress

Links: Western University primary reporting · Peer-reviewed paper in Neuro-Oncology Advances · MedicalXpress coverage

Worth catching up on:

4. Stanford team maps how gliomas hijack a normal brain-repair signal

This research falls just outside this week’s formal 9–16 August window, because the underlying Nature Neuroscience paper was published on 6 August. It attracted further reporting during this week, however, and is sufficiently interesting to merit a brief catch-up.

Why it matters

It isn’t a new discovery that active neurons can encourage glioma growth. Professor Michelle Monje’s Stanford laboratory has spent close to a decade building the evidence that brain tumours exploit normal neuronal activity.

What is new here is a much more precise molecular chain of events, potentially giving drug developers specific points in the pathway that might eventually be targeted.

The study shows that NLGN3 - neuroligin-3 - a protein released by active nerve cells, binds directly to a receptor called CSPG4 on glioma cells and on healthy oligodendrocyte precursor cells.

That binding triggers CSPG4 to be cut from the cell surface by an enzyme called ADAM10.

This changes tension in the cell membrane and activates PIEZO1, a mechanical sensor.

In healthy precursor cells the pathway helps maintain a youthful, replenishing state.

Glioma appears to hijack the same mechanism, using it to keep tumour cells dividing and invading.

Researchers used patient-derived cancer-cell membranes and an NLGN3-affinity system to identify CSPG4 as the binding partner.

Using CRISPR-Cas9 to remove either CSPG4 or PIEZO1 from human glioma cells before implanting them into mice sharply reduced tumour growth.

Removing NLGN3 itself from mice reduced healthy oligodendrocyte precursor-cell numbers by around 30%, reinforcing the idea that the pathway performs a genuine function in the healthy brain.

Caveat

This remains laboratory science involving mouse models and patient-derived cells and tissues. There is currently no treatment targeting this particular pathway.

The central challenge will be whether researchers can interfere with the tumour’s use of the pathway without also damaging the normal repair function that the brain itself needs.

Originator: Professor Michelle Monje and team, Stanford University School of Medicine; first author Yoon Seok Kim

Clinical Sponsor: Not applicable - preclinical laboratory research

Funder: Specific grant funding not detailed in the material available for this edition

Campaigning Organisation: Not applicable

Reporting Source: Nature Neuroscience; subsequent coverage including MedicalXpress

Links: Nature Neuroscience paper · PubMed · MedicalXpress coverage

That’s all for this week

A quiet seven days, but with an interesting common thread: finding new ways to reach the tumour.

NEO100 attempts to approach the brain through the nose. ChemoSeed puts chemotherapy directly into the surgical cavity. Intratumoral Modulation Therapy places the therapeutic device inside the brain itself.

Different technologies, different stages of development - but all confronting one of the central problems that has frustrated brain-tumour treatment for decades:

It isn’t enough to have a treatment that can kill tumour cells. Somehow, you have to deliver it.

What follows is a case for what incremental investment could realistically achieve - and how we’d know it was working long before anyone had to wait a decade to find out.

Introduction, in plain English, before the detail

• This is not a claim that a cure is coming. It is a costed case for speeding up research that’s already under way.

• The ask is for £12m a year, (as per BTNL special edition of 25th July - ‘Whose numbers are these, anyway?’), to be spent in stages - not as one lump sum with a decade-long wait to find out if it worked.

• What it buys is a shared framework within which patients are matched to the right trial faster, and several drugs are tested simultaneously instead of one at a time.

• We know this approach works because it already has done so. Drugs picked this way have reached patients two to four years faster than the old approach, in a peer-reviewed study of 63 real FDA approvals.

• One catch: this doesn’t speed up the treatment of every tumour equally because a slow-growing tumour still takes time to show whether a treatment worked, regardless of the method used to choose the most appropriate drug.

• The good news is that even a cautious 5–10% improvement would be worth several times the £12m investment - see the chart below.

The rest of this article sets out the evidence and the caveats behind each of these points.

Where we left off

In the last special edition on BTNL (‘Cost of Deaths of Under-45s’ 6.8.26) we ended on a question: ‘Do we keep paying for loss, or start investing properly in research acceleration?’1 That’s the right question, but it invites an obvious follow-up. An overnight cure isn’t realistic, and nobody seriously claims otherwise. So what, specifically, would ‘investing properly in research acceleration’ actually buy - and how would we know it was working before a decade had passed?

This article tries to answer that honestly: firstly by explaining the framework, then with a real example of how much that framework can compress a timeline, and finally with a staged way of spending that produces checkable evidence long before any long-term survival data matures.

How long does the journey from lab to patient really take?

Across all disease areas, it takes an estimated 8.5 years on average for a drug to go from early laboratory discovery to approval for human use, according to FDA-based analysis.2 Oncology specifically tends to sit at the longer end of that range: the traditional pathway for a cancer drug is often quoted at up to approximately 15 years from discovery to approval.3 This is the baseline research acceleration is trying to compress - not eliminate, compress.

The real evidence that this timeline is compressible

This isn’t a hopeful guess. There’s a specific, peer‑reviewed, and directly relevant precedent for how much a smarter development strategy can save.

A 2016 analysis in the journal Oncotarget examined all 63 cancer drugs approved by the FDA between 1998 and 2014, comparing those developed using a personalised, biomarker‑driven strategy against those that weren’t.4 A biomarker-driven strategy is an approach to diagnosing, classifying, and treating brain tumours based on the tumour’s molecular and genetic characteristics. For example, vorasidenib and safusidenib are used specifically to treat tumours which have certain gene mutations. Another example is niraparib, which is used to attack tumours which are shown by tests to have a particular vulnerability to that drug known as ‘synthetic lethality’.

Putting this another way, there are over 100 different types of brain tumour, so ‘one-size-fits-all’ was never going to work. Biomarker-driven treatment is the opposite to ‘one-size-fits-all’; it is personalised and targeted, and based on biochemical analysis of the individual tumour.

Biomarker‑driven drugs reached approval in a median of 64.6 months of total clinical development, against 87.1 months for non‑personalised targeted drugs and 112.7 months for traditional chemotherapy‑style drugs. That’s roughly 1.9 years faster than the non‑personalised norm, and roughly 4 years faster than the traditional chemotherapy route. Individual examples in that same dataset make the point vividly: imatinib reached approval in 36 months, ceritinib in 42.7 months (approved after Phase I alone), and dabrafenib in 47.1 months - all biomarker‑driven, all dramatically faster than the 87–113 month norm.

This matters for brain tumours specifically, because vorasidenib, dordaviprone and safusidenib are all developed via exactly this kind of biomarker‑driven, targeted approach.

The honest complication

Vorasidenib itself took nine years to develop - near the slow end of the dataset, not the fast end - despite being biomarker-driven by every definition. That’s because grade 2 IDH-mutant glioma is naturally slow-growing, and its trial’s primary measure (progression-free survival) could only mature as fast as progression events actually occurred. The method didn’t fail; it simply couldn’t outrun the biology. Dordaviprone shows the other side: because its trial could use a faster surrogate measure (tumour response, not survival), it reached patients through accelerated approval instead. Biomarker-driven development still compresses everything it can reach - smaller trials, less time wasted on patients unlikely to respond, faster regulatory review once efficacy is shown. It just can’t compress how long a slow-growing tumour takes to show its hand. However, although you can’t compress biology, you certainly can compress bureaucracy, and that’s where the framework, or infrastructure, that we are advocating comes into play.

What sustained investment could buy more of is this infrastructure that makes biomarker‑driven development possible at scale, rather than one drug at a time:

• Molecular‑matching infrastructure - identifying which patients carry which biomarker, faster. The Tessa Jowell BRAIN‑MATRIX programme (£2.8m) is a live UK example.5

• Platform and adaptive trial capacity - testing multiple biomarker‑matched drugs against shared infrastructure and a shared control, rather than building a new trial from scratch for each one. The 5G platform trial for glioblastoma (£3.36m, Cancer Research UK and the Minderoo Foundation) is a live UK example.6

• Faster patient recruitment. An independent analysis by the contract research organisation IQVIA of decentralised trial elements found substantial reductions in time from final protocol to first patient enrolled, alongside fewer protocol deviations, compared with traditional trial delivery.7

• Better on‑treatment biomarkers, to support faster, evidence‑based decisions about what’s working. The 21 July 2026 Neuro‑Oncology paper on early MRI changes predicting dordaviprone response is a live, current example of exactly this kind of finding emerging in real time.8

A staged, incremental way forward, as opposed to a single, big lump sum request

The £12m/year accelerant‑infrastructure figure already modelled in our special edition of 25^th July (‘Whose numbers are these, anyway?’) does not need to be spent, or asked for, in one lump sum with a decade‑long wait for results. It can be structured in stages, each with milestones that are checkable years before any survival data matures.

It is not a payment to compress one candidate’s existing timeline through added parallel effort, in the way that the first two articles in this series describe (The Ten Year Gap’ 10.7.26 and ‘What’s a Year Worth?’ 15.7.26); it is an investment in shared, reusable infrastructure - molecular matching, platform‑trial capacity - that lets many future biomarker‑driven candidates benefit from the kind of acceleration the Oncotarget evidence above describes, rather than each one paying its own crashing costs from scratch.

Stage 1 (Years 1–2): build the infrastructure.
Centres onboarded to molecular‑matching capability; number of biomarker‑matched trial arms opened; a measured baseline time‑to‑first‑patient, with an explicit target for reducing it.

Stage 2 (Years 3–5): prove the process is faster.
Measured reduction in average time‑to‑trial‑decision across the portfolio; number of treatments reaching regulatory submission via a biomarker‑driven, accelerated pathway, benchmarked against the aforementioned parameters derived from the Oncotarget analysis.

Stage 3 (ongoing): the payoff matures.
Treatments reach NHS patients measurably faster than the historical baseline. This is the stage at which the QALY and exchequer modelling below stops being illustrative and starts being checkable against real approval dates.

This structure means a funder never has to take it entirely on faith. Stage 1 and Stage 2 produce evidence of progress within two to five years - long before anyone needs to wait a decade to find out if the investment worked.

What would this be worth, if it works?

To be clear about what is and isn’t being claimed here: this is not a projection that any specific new drug will arrive, or that any specific number of lives will be saved. It’s the same illustrative approach used throughout this scrutiny - asking what a conservative, stated percentage improvement would be worth - now applied through the QALY lens established for the under‑45 cohort specifically, rather than the whole‑population financial burden used in the third article of this series (as per ‘Whose numbers are these, anyway?’). These are two different lenses on the same underlying assumption, not two separate effects to add together.

Illustrative acceleration effect

The chart above shows an illustrative 5–15% reduction in the under 45 cohort’s annual burden, applied to both the £638.88m QALY value figure and the £195.3m exchequer facing figure established in our fourth article, ‘Cost of Deaths of Under-45s’ (6th August), compared against the same £12m/year accelerant infrastructure investment.9

Read honestly, this chart says something important: through the narrowest, most cash‑focused lens - this cohort alone, exchequer terms only - a 5% effect doesn’t quite clear the cost of the investment (0.8×). The case only becomes comfortably positive in cash terms once the effect reaches roughly 10%. However, through the QALY lens, even a 5% effect clears the investment nearly threefold, because health‑value loss is simply larger than cash cost for a group this young. And considered across the whole brain tumour population, rather than under‑45s alone, the same conservative 5–10% assumption already produced a 21–43× return. None of these figures contradict each other. They’re different, honestly‑labelled slices of the same argument, and the argument holds up under all of them - it’s simply strongest through the widest lens and tightest at the narrowest.

How will we know if it’s working?

The staged milestones above are the answer to this question. Long before any QALY or exchequer figure can be checked against reality, these can be:

• Time‑to‑first‑patient for new biomarker‑matched trials, measured against the current baseline.

• Time‑to‑trial‑decision across the portfolio, benchmarked against the approximately 65–113 month range established in the Oncotarget analysis above.

• Number of biomarker‑matched trial arms open at any one time, as a direct measure of infrastructure capacity.

• Number of treatments reaching regulatory submission via an accelerated, biomarker‑driven pathway.

None of these require waiting for a single patient’s outcome. All of them are checkable within the first few years of a staged investment - which is exactly what a sceptical funder, or a sceptical reader, should ask for before backing the contention made by this article.

The bottom line

Accelerating brain tumour research from an incremental funding base isn’t a leap of faith. The framework - biomarker‑driven development - already has a peer‑reviewed paper trail showing it cuts roughly two to four years off the historical norm. Investing further in the infrastructure that makes that framework scale - matching, platform trials, faster recruitment, better on‑treatment biomarkers - is risk‑managed with checkpoints, not a bet on a wing and a prayer.

An overnight cure was never the honest target. But a staged, checkable acceleration of a process that has already been shown to compress time is eminently achievable.

Links and citations

Contents:

1. Novel Therapeutics Accelerator

2. Nuvation Bio confirms safusidenib expansion

3. Light-activated nanoparticle targets glioblastoma

4. Renewal of funding for preclinical CAR-phagocyte research in paediatric high-grade gliomas

1. Brain Tumour Research invests £516,000 in the Novel Therapeutics Accelerator

Summary: Brain Tumour Research has announced a further £516,000 over three years for the Brain Tumour Research Novel Therapeutics Accelerator programme, run with the Tessa Jowell Brain Cancer Mission.

The programme is not a treatment or clinical trial itself. Instead, it gives academic and industry teams expert help with the difficult translational steps between laboratory discovery and clinical testing.

Brain Tumour Research says the accelerator has reviewed 22 innovative therapies since launch, with projects going on to secure more than £70m in follow-on funding. One reviewed therapy is preparing for first-in-human testing, and another has already treated its first patient.

Why it matters: This is a concrete UK example of research-acceleration infrastructure - the kind of motorway that should help promising therapies move faster towards the clinical trial stage.

It tackles the “valley of death” problem: promising brain tumour treatments often fail not because the idea is no good, but because the translational path from the laboratory stage to clinical testing is under-supported.

Originator: Brain Tumour Research / Tessa Jowell Brain Cancer Mission
Clinical Sponsor: N/A — accelerator/advisory programme, not a clinical trial
Funder: Brain Tumour Research
Campaigning Organisation: Brain Tumour Research; Tessa Jowell Brain Cancer Mission
Reporting Source: Brain Tumour Research, 3 August 2026
Link: Brain Tumour Research announcement

2. Nuvation Bio confirms safusidenib expansion and reports strong balance sheet to fund it

Summary: This is not a repeat of our recent safusidenib special edition. It is a financial follow-up: confirmation that Nuvation Bio has the cash to run the expanded safusidenib programme it has now announced.

Nuvation Bio’s Q2 2026 results, released on 6 August, reaffirmed the safusidenib programme expansion: a pivotal Phase 3 trial in newly diagnosed Grade 2 IDH1-mutant glioma outside the US, plus a new Phase 2 study for patients who progress after vorasidenib.

The company reported $661 million in cash and marketable securities as of 30 June 2026, plus a further $36.5 million secured in July from its ‘convertible notes’ overallotment.

Why it matters: Drug-development announcements are one thing; having the financial muscle to carry them through is another. This update gives some reassurance that the expanded safusidenib glioma programme is not just scientifically planned, but also financially backed.

Originator: Nuvation Bio Inc.
Clinical Sponsor: Nuvation Bio Inc.
Funder: Nuvation Bio Inc.
Campaigning Organisation: Not applicable
Reporting Source: PR Newswire / Nuvation Bio Investor Relations, 6 August 2026
Link: Q2 2026 results

3. Light-activated nanoparticle “double punch” targets glioblastoma surgery and recurrence

Summary: Researchers from the University of Technology Sydney, Harvard and Henan universities have reported a light-activated nanoparticle platform designed to do two jobs: help surgeons see glioblastoma more clearly during surgery, then help destroy cancer cells left behind afterwards.

The UTS report says the platform was published in Science Translational Medicine and, in mouse models, suppressed recurrence, with 100% survival at 60 days in treated animals compared with 42 days after surgery alone.

The researchers themselves stress that this is still early-stage mouse-model work and that performance must be confirmed at the scale of a human brain.

Why it matters: The appeal is obvious: better tumour visibility during surgery plus a “clean-up” effect afterwards. But the caveat is equally important: this is not yet a patient-ready treatment.

Originator: Professor Bingyang Shi and colleagues, University of Technology Sydney / Harvard / Henan collaborators
Clinical Sponsor: N/A — preclinical research, not a human trial
Funder: Not confirmed in the accessible UTS report
Campaigning Organisation: None identified
Reporting Source: UTS News; Science Translational Medicine
Links: UTS press release / Phys.org coverage

4. Ohio State identifies SET/PP2A pathway as a possible way to make GBM more vulnerable to radiotherapy and chemotherapy

Summary: Research from The Ohio State University Comprehensive Cancer Center - James Cancer Hospital points to SET and related PP2A-blocking proteins as possible treatment-sensitisation targets in glioblastoma.

The work, published in Cancer Letters, suggests glioblastoma cells use proteins including SET, ANP32A and CIP2A to suppress PP2A, an enzyme involved in controlling growth, survival and DNA-damage repair signals.

In laboratory and animal models, blocking these proteins reduced cancer-cell survival and increased sensitivity to radiation.

Why it matters: This is not a new patient treatment. It is preclinical research. But it is relevant because it is aimed at making existing treatments - radiotherapy and chemotherapy - work better against glioblastoma.

Caveat: The report is explicit that the findings are preliminary, have not been tested in patients, and should not lead to off-label self-medication.

Originator: John Ryan Jacob, Arnab Chakravarti, Kamalakannan Palanichamy and OSUCCC–James colleagues
Clinical Sponsor: N/A - preclinical/translational research, not a clinical trial
Funder: Not confirmed in the accessible report
Campaigning Organisation: None identified
Reporting Source: Ohio State University Medical Center / MedicalXpress; Cancer Letters
Link: MedicalXpress coverage

5. NIH renews funding for preclinical CAR-phagocyte research in paediatric high-grade gliomas

Summary: The University of Colorado Cancer Center has reportedly secured a $1.28m NIH R01 renewal to continue developing CAR-engineered macrophages/microglia targeting B7-H3 in paediatric high-grade gliomas, including glioblastoma and DIPG.

The strategy combines CAR-phagocytes with CD47 blockade, aiming to overcome one of cancer’s immune-evasion mechanisms.

Why it matters: This is a grant-watch item rather than a treatment announcement. It offers an alternative angle to CAR-T therapy, which has struggled in solid tumours partly because of tumour penetration and the suppressive tumour microenvironment.

The work remains preclinical, but it reflects sustained National Cancer Institute investment in cellular immunotherapy approaches for paediatric brain tumours.

Caveat: This item is based on secondary reporting and grant information rather than a full University of Colorado press release. It should therefore be treated as a funding-watch / preclinical research item, not evidence that a patient-ready therapy is available.

Originator: University of Colorado Cancer Center; Dr Siddhartha S. Mitra
Clinical Sponsor: N/A — preclinical research
Funder: National Cancer Institute, NIH R01
Campaigning Organisation: None identified
Reporting Source: AllSci News, citing NIH RePORTER grant information
Link: AllSci News: University of Colorado renews work on CAR-phagocyte paediatric brain cancer therapy

That’s all we have for this week. Please, if anyone has anything to add, or any ideas of how to improve this substack – just shout! (munrohugh@aol.com)

Try putting a price on 484 deaths a year

Impossible, of course. But government, health economists and public bodies do try to put numbers around loss, because without numbers policy decisions become almost impossible to argue about.

Used carefully, those numbers can help. Used carelessly, they can mislead.

This article is about the difference between two very different questions.

The first is: what is lost to society when hundreds of young people die of brain tumours each year?

The second is: what might be saved or retained by the public finances if those deaths were prevented?

Those are not the same question. They do not produce the same answer. And mixing them up is one of the easiest mistakes to make when arguing for investment in this disease.

The number behind the number

Every year, an average of 484 people aged 44 and under die from brain, other central nervous system (CNS) and intracranial tumours in the UK.¹

That figure comes from BTNL’s calculation using Cancer Research UK’s Cancer Stats Data Hub mortality breakdowns for brain, other CNS and intracranial tumours, summing the 0–44 age bands and averaging across the 2022–2024 period.¹

The estimated median age at death within this group is approximately 37, based on the grouped five-year age bands. In plain English, most of these are people who, on any ordinary measure, still had decades of life ahead of them.^1,2

So, what do those deaths cost?

There are two honest answers.

One is about the value of health and life itself. The other is about consequential financial costs that might affect public finances.

What a NICE-threshold-based illustration says about the value of the loss (NICE is the National Institute for Health and Care Excellence).

Start with an established public-sector cost-effectiveness framework.

UK period life expectancy at birth in 2022–2024 was 83.0 years for females and 79.1 years for males-about 81 years as a rounded proxy. Subtract the estimated median age at death of 37, and each death represents around 44 future life-years in this simple calculation.^2,4

Multiply that by 484 deaths a year, and the result is:

21,296 undiscounted future life-years lost by each annual death cohort^1,2,4

For an illustrative upper-bound calculation only, assume that each remaining life-year is equivalent to one quality-adjusted life-year (QALY) and value it at £30,000-the midpoint of NICE’s current £25,000–£35,000 cost-effectiveness threshold. On that assumption, the deaths represent:³

£638,880,000 in undiscounted lifetime health value associated with each annual death cohort^1–4

A more technically precise version of the same illustration, using ONS (Office for National Statistics) age-specific remaining life expectancy and the CRUK grouped age bands, puts the figure closer to 21,809 undiscounted future life-years, or approximately:^1,2,4

£654.3 million for each annual death cohort under the same upper-bound assumption^1,2,3,4

Either way, the order of magnitude is the same.

But this is where the distinction matters.

Would a cure free up £638.88 million to spend elsewhere?

No.

That is the part worth being completely honest about, because it is exactly the sort of claim that gets challenged the moment it reaches a policy audience.

The NICE range is not a sum of money anyone pays or receives. It is not cash sitting in the Treasury. It is not a line in an NHS budget.

It is a cost-effectiveness decision rule: a way of judging whether the additional health generated by a treatment justifies its additional cost within a constrained NHS budget.³

Multiplying undiscounted life-years by the £30,000 midpoint can therefore provide only an illustrative upper-bound health-value proxy. It is not a formal QALY analysis and it is not a fiscal saving.³

A miraculous overnight cure would not hand the Treasury a cheque for £638.88 million.

It would give 484 families a year more decades with someone they love.

That is real - arguably more real than any figure in this article - but it is not the same as public money being freed up for hospitals, schools or defence.

This is the same distinction drawn in ‘Whose Number Is It Anyway?’: the value of health and life lost sits apart from harder-edged financial costs such as lost earnings, welfare payments, NHS spending and unpaid care.

The £638.88m upper-bound figure sits in the first category. To ask what might be at stake for the public finances, we need to build a separate estimate for this specific under-45 cohort.

What might be at stake for the public finances?

This section is deliberately more cautious.

It does not claim to be an audited Treasury appraisal. It is an illustrative gross fiscal-exposure estimate, using transparent assumptions.

The aim is to ask: if a person under 45 dies of a brain tumour rather than living through a full working life, what kinds of public revenue or public spending are most directly affected?

Three components can be estimated. A fourth is deliberately excluded from the headline.

1. Lost tax and National Insurance revenue

This is the largest component, because this cohort dies young enough for many working years to be lost.

Using an estimated median age at death of 37 and the legislated State Pension age of 68 for people born after 6 April 1978 gives around 31 potential working years lost per death.^2,7

Using median UK full-time gross annual earnings of £39,039, and 2026/27 income-tax and National Insurance rates (including both employee and employer contributions):^5,6

• income tax: £5,293.80 per year

• employee National Insurance: £2,117.52 per year

• employer National Insurance: £5,105.85 per year

That gives a combined annual income-tax and NI figure of £12,517.17.^5,6

Over 31 potential working years, that is:

£388,032 per person

Scaled to 484 deaths in one year, the figure is approximately:

£187.8 million in undiscounted gross lifetime revenue associated with each annual death cohort^1,2,5–7

This is not a perfect measure. It assumes full-time median earnings, continuous work to State Pension age, no discounting, no career progression, no employment gaps and no replacement of lost labour by other workers. Some assumptions may overstate the figure; others may understate it.

But as a transparent estimate of the order of magnitude, it is useful.

2. Welfare costs

The Brain Tumour Charity’s York Health Economics Consortium report (the same source behind the £18.7bn / £5.17bn figures used elsewhere in this series) estimates UK-wide welfare-system costs of £357.8 million across roughly 12,700 annual primary brain-tumour diagnoses of all ages.⁸

That works out at about:

£28,172 per diagnosis⁸

If we use this all-ages figure as a simple proportional basis for the 484 under-45 deaths, it produces an attributed figure of:

£13.6 million^1,8

This may understate or overstate the true welfare cost for this group, because younger fatal cases are unlikely to have the same benefit profile as the average diagnosis. Nor can it be treated as a saving caused solely by preventing death: some costs might persist, or even rise, if treatment extended life with disability. It is best understood as a proportional illustration using the available data.

3. Direct NHS treatment costs (excluded from the headline figure)

The same report gives direct healthcare costs of £287.6 million across roughly 12,700 diagnoses.⁸

That is about:

£22,643 per diagnosis⁸

Proportionately attributed to 484 deaths, that would be around:

£11.0 million^1,8

This is real health-system expenditure, but it is not the same as cash being freed for the Treasury. Successful treatment could also increase lifetime healthcare use for some patients, so the figure should not be treated as an automatic saving.

If these deaths were prevented, NHS capacity and resources would not simply disappear into a public bank account. Capacity released in one place could be used to treat other patients; additional survival might require care elsewhere. This is the opportunity-cost logic that underpins NICE’s assessment framework.³

The figure is therefore shown for completeness but excluded from the headline gross fiscal-exposure estimate.

4. Unpaid care costs (real, but not a government bill)

Unpaid care is a genuine economic loss to families and society. It matters deeply.

But it is not currently a bill the government pays in full. So, it is not money that would be ‘freed up’ for the Treasury if a cure arrived tomorrow.

For that reason, it is excluded from the headline, even though it remains part of the wider economic burden.

The headline estimate

Putting the two most clearly Exchequer-facing components together-undiscounted gross income-tax and National Insurance revenue under the full-time median-earnings assumption, plus the proportionally attributed welfare-system cost-gives an illustrative exposure of about:

£201.4 million in undiscounted gross lifetime fiscal exposure associated with each annual death cohort^1,2,5–8

Adding the separately identified NHS treatment-cost estimate would take the total to around:

£212.4 million^1,2,5–8

But that is a different kind of number, because NHS costs are better understood as health-system resources that may be reallocated or changed, not cash released directly to the Treasury.

So the cleaner headline answer to the question is:

Approximately £201.4 million in illustrative gross lifetime fiscal exposure for each annual death count.

Not an annual Treasury saving. Not a net fiscal benefit. Not a guaranteed cash release. But a serious, transparent illustration of the public-finance scale involved.

To summarise

£638.88 million is one transparent upper-bound answer to the question: what health value is lost when 484 people aged 44 and under die of brain tumours in one year, if every remaining life-year is treated as one QALY and valued at £30,000?^1–4

Using more precise age-specific remaining life expectancy, the answer may be closer to £654.3 million.^1,2,3,4

That is a useful figure for making the moral, social and health-value case for investment, provided it is described as an illustrative upper bound rather than a cash saving or formal QALY appraisal.

£201.4 million is a different kind of number. It is an illustrative estimate of undiscounted gross lifetime fiscal exposure associated with each annual cohort: income-tax and National Insurance revenue that may never be collected, plus proportionally attributed welfare-system costs, under clearly stated assumptions.^1,2,5–8

It is smaller. It is more limited. It is not an annual cash loss, and it comes with substantial caveats.

But that does not weaken the argument. It strengthens it.

Because a serious campaign should know the difference between health-value loss and Treasury-facing cost. A policy audience will expect that distinction. Advocates should expect it of themselves.

The deaths of under-45s from brain tumours are not just tragic. They are measurable. Each annual cohort represents hundreds of millions of pounds in illustrative lifetime health-value loss and, under this model, around £201 million in undiscounted gross lifetime public-finance exposure.^1–8

Those are different numbers.

Both matter.

And both point in the same direction: Britain is already paying heavily for slow progress against brain tumours. The question is whether we keep paying for loss, or do we start investing properly in research acceleration?

Notes on the numbers, for anyone who wants to check them

1. 484 average annual UK deaths aged 44 and under from brain, other CNS and intracranial tumours, 2022–2024: BTNL calculation from Cancer Research UK Cancer Stats Data Hub mortality breakdowns.

2. Estimated median age at death of approximately 37: BTNL estimate from grouped five-year CRUK age bands.

3. NICE’s current £25,000–£35,000 per-QALY threshold: NICE, ‘Changes to NICE’s cost-effectiveness thresholds take effect’ (2 April 2026); policy background: UK Government, ‘Landmark UK–US pharmaceuticals deal’ (1 December 2025).

4. UK life expectancy at birth, around 81 years, and age-specific remaining life expectancy at age 37: Office for National Statistics national life tables.

5. Median UK full-time gross annual earnings, £39,039, April 2025: ONS Annual Survey of Hours and Earnings.

6. Income tax, Personal Allowance and National Insurance rates for 2026/27: House of Commons Library, ‘Direct taxes: rates and allowances for 2026/27.’

7. State Pension age applicable to a person now aged about 37: 68 under the current legislated timetable for people born after 6 April 1978. Department for Work and Pensions, ‘State Pension age review 2023’.

8. Welfare and direct healthcare cost figures: The Brain Tumour Charity / York Health Economics Consortium, ‘The Cost of a Brain Tumour: The Economic Case for Urgent Action’ - £357.8m welfare costs and £287.6m direct healthcare costs across roughly 12,700 annual diagnoses.

Methodological note: This article is an illustrative model, not a formal Treasury appraisal. Figures derived from each annual cohort represent undiscounted lifetime quantities, not cash lost or released in a single year. Its purpose is to separate different kinds of value clearly enough that the argument can be checked, challenged and improved.

Archie Goodburn pleads on BBC breakfast television for more funding for rare cancer research

By George O’Neill, BBC Sport Scotland, Published 4 August 2026, 10:43 BST

Prime Minister Andy Burnham has responded to Archie Goodburn’s impassioned plea for more money and resources to be put into brain cancer research by inviting the Scottish swimmer to a personal meeting.

Tears rolling down his face, the 25-year-old had implored “give us a chance to fight for our lives” while speaking about the subject on BBC Breakfast.

Link: Watch Archie’s plea here.

Replying to the video on TikTok, Burnham said: “Archie, thank you for this. I know how hard it must have been.

“What you’re doing for other people while you’re going through all this yourself shows the best of us.

“I’d really like to meet you and hear from you directly and I’ve asked my team to get in touch.”

Goodburn was diagnosed with inoperable brain tumours just over two years ago and has since passionately advocated for an increased focus on the disease.

“One of my dreams is that we see a future where brain cancer isn’t the biggest killer of patients under 40,” he said during the Commonwealth Games in Glasgow.

And, making a direct appeal to Downing Street to help ease the “unbearable” suffering of brain cancer patients and their families, he added: “Andy Burnham, if you believe and want to make a change and a difference to the future of our country and our young population, please listen to us.

“Please establish a national brain cancer lead and give us a chance to fight for our lives because the things that patients are going through out there are unbelievable and so unjust.

“There are clinical trials around the world, in the US, in Australia, in China, that could change lives. We are not seeing them in the UK and it is unfair and unjust.

“Please invest and make the difference. I’m on my knees begging for - not just my own life - but for those of my best friends and those that are affected and the families around the country.

“This has to change. It’s been going for too long and the suffering is unbearable.”

• ‘Medal dream didn’t come true but my other dream is fighting brain cancer’

  • Published

    27 July

• The Commonwealth Games swimmer who might not see 40

  • Published

    25 June 2025

The Rare Cancers Bill - led by Scott Arthur MP - became law earlier this year.

It aims to incentivise research and investment into the treatment of rare types of cancer, but Goodburn says it does not go far enough.

“It’s a step in the right direction but it’s not going to make the changes that we need,” he said. “I’m extremely grateful for Dr Scott Arthur and the work he’s done there.

“The rare cancers bill covers 14 different types of rare cancers. Brain cancer is one of these in terms of incidents of diagnosis, but it’s not the same in terms of the number of deaths it causes.

“Yes it’s rare, but it’s extremely deadly. This frustrates me immensely.

“The rare cancers bill establishes two new positions: a clinical lead and a research lead. These two roles are part-time across 14 different types of cancers and they accumulate to 36 days a year.

“There’s two roles to cover 14 different types of cancer and change the landscape of treatment.

“I do not see how 36 days a year is going to make that change for 14 different types of cancers, let alone the most deadly type of cancer.”

‘My dream is a future for brain cancer patients’

The Commonwealth Games in Glasgow were a huge driving force for Goodburn, amid the emotional turmoil of the past two years, and he completed his goal of reaching the 50m breaststroke final.

He missed out on the medal he craved, but even after that disappointment he was able to eloquently and passionately advocate for increased brain cancer research.

Few athletes - if any - at the Games got a bigger ovation than Goodburn.

“It was an honour to compete in front of such a fantastic and supportive home crowd,” he said, reflecting on the Games and his post-race interviews.

“To see the support off the back of those interviews has been really, really touching and inspiring.

“In that moment I was speaking about what felt important to me and the crowd were very supportive of my journey, and it only felt right to address that when I got out the pool.

“What I do in the pool is only possible because of the medical advances that have been made and the campaign that’s been done by the whole community. It was really a chance to give back and make sure that momentum keeps going.”

And he insists he will continue to try to make a difference for other patients.

“I partly feel like why we don’t hear so much about brain cancer and why the awareness is so poor is because patients unfortunately die so quickly,” he said.

“I have this prognosis which is slightly longer and it’s given me this time to advocate - it’s two years since I was diagnosed.

“I think when you’re really backed up against a wall like that there’s only really one way to go and that’s to go and fight.” Archie Goodburn 4.8.26

Dr Sophie Morse, Assistant Professor of Bioengineering at Imperial College London, leads research into the use of low-intensity focused ultrasound (FUS) as a non-invasive means of treating brain disease. Her brain tumour programme is investigating both improved drug delivery and the possibility that ultrasound itself may beneficially alter the activity of immune cells within the tumour environment. A major current project, funded by the Astro Brain Tumour Fund, is testing treatments against different low-grade glioma subtypes and examining whether FUS can enhance the delivery of drugs including vorasidenib and niraparib in preclinical models. The longer-term aim is to develop personalised combinations capable of controlling low-grade tumours and potentially delaying or preventing their progression to higher-grade disease. The work remains preclinical, but it addresses one of neuro-oncology’s greatest obstacles: getting an effective concentration of treatment through the blood-brain barrier without damaging surrounding healthy brain tissue.

On 23 July 2026, I met Dr Morse to ask how this work might eventually reach patients and what stands in its way. The following questions and answers have been reviewed and authorised by Dr Morse prior to publication.

HM: Does the BBB-penetration limitation apply more to niraparib than to vorasidenib in this combination - or to both? I’ve read that vorasidenib is sufficiently BBB-penetrative and doesn’t need FUS to get through the barrier.

SM: Both drugs, because of their small size and properties, have inherently good blood-brain barrier penetration. The extent of their penetration could be enhanced even further by FUS in a targeted way, with the main advantage being that this could lower the drug dose that needs to be taken by patients, to reduce side effects.

HM: Will the FUS-assisted drug-delivery programme be run in parallel with, and in addition to, the daily oral intake of vorasidenib? Or will FUS have to be the sole method of delivery?

SM: There’s lots of figuring out to do. We envisage that in the early stages patients would continue with the daily oral intake, supplemented with, say, a monthly FUS-assisted session to get other larger synergistic drugs to fight the tumour in a complementary way.

HM: Could the niraparib plus FUS combination be given alongside first-line oral vorasidenib, or do you see it as a potential second-line option for patients who progress on vorasidenib alone? In other words, could we have oral ‘vora’ with FUS-delivered ‘nira’?

SM: Yes, certainly - this could be one of many options which ongoing research is exploring. The ultrasound, we hope, would reduce the side effects of niraparib.

Editor’s note: The proposed mechanism is targeted delivery: if FUS increases the amount of a drug reaching the tumour, a lower overall dose may be sufficient, potentially reducing systemic side effects. This remains a research hypothesis to be tested.

HM: When do you think you will be doing clinical, human trials of FUS plus vorasidenib, with or without niraparib? Could you give me an optimistic, realistic and pessimistic estimate for moving from the current preclinical phase to a first human study?

SM: Difficult to say. These things take such a long time, normally because of ethics paperwork and limited funding. The good thing is that, as long as there is funding to run a trial, the current trial being run by the team at St Mary’s Hospital using focused ultrasound in patients with glioblastoma should speed up being able to do this in low-grade gliomas. We need to figure out which drugs would be best for the clinicians to test in low-grade gliomas, which is what we are working on at the moment in the lab. But if funding is available, their high-grade results would speed up low-grade clinical work.

[Editor’s note: The recruiting St Mary’s study, ISRCTN14636207, is a Phase I trial of sonodynamic therapy in glioblastoma. Patients receive oral 5-aminolevulinic acid (5‑ALA), which accumulates preferentially in tumour cells, before MRI-guided focused ultrasound is applied. It therefore uses FUS to activate a tumour-selective agent rather than primarily to open the blood-brain barrier. Its safety, regulatory and practical experience may nevertheless help inform later brain tumour FUS trials.]

HM: You’ve mentioned that you’re impeded by a combination of funding scarcity and bureaucracy - what do you suggest is done about the latter? In other words, which delays are genuinely reducible with money, and which are fixed because of biology?

SM: One of the difficulties I face is the time I use up writing grant applications, submitting them and then waiting for responses, which can take months and even up to a year or more from start to end. That process is disproportionately long and acceptance rates for most of these grants are generally 10–15%. It’s frustrating to spend so much time writing these long applications in a good way to have so many rejected. It ends up putting you off from starting these applications in the first place, unless one has plenty of time - which is almost never the case. My time would be much better spent guiding my team than doing all this writing.

HM: Has anyone associated with the new NIHR National Specialty Lead for Rare Cancers role - created under the Rare Cancers Act 2026 - contacted you? Are you aware of who, if anyone, has been appointed?

SM: No, I’ve never heard of this - what is it?

Dr Morse’s reply establishes that she has not been contacted and was unaware of the role; it does not tell us whether other researchers have been approached. At the time of writing, on 3 August 2026, I could find no public announcement naming the appointee. The Government has said that the NIHR National Specialty Lead for Rare Cancers will be a part-time appointment based within the NIHR Research Delivery Network, providing strategic oversight of rare-cancer studies and addressing obstacles to research delivery. The post is distinct from the separate National Clinical Lead for Rare Cancers promised in the National Cancer Plan. The real test will be whether these new structures become visible, accessible and useful to researchers working at laboratory level.

Links and further reading

• The Morse Lab

• St Mary’s sonodynamic therapy trial - ISRCTN14636207

• Government answer on the NIHR National Specialty Lead for Rare Cancers

• National Cancer Plan for England - rare cancers and research

• All-Party Parliamentary Group on Brain Tumours

What I took from the meeting

I came away from the meeting enlightened and enthusiastic about the potential of focused ultrasound. I was also struck by how much faster promising research might progress if scientists had greater access to specialist support with grant applications. Researchers like Dr Morse have teams to supervise, experiments to design, students to teach and administrative responsibilities that cannot be avoided. Dedicated research-development professionals could help identify suitable funding opportunities, manage budgets and documentation, and shape applications effectively, while leaving scientists to do the science.

Such support would not remove the need for competition, peer review, ethical approval or rigorous safety testing - nor should it. These safeguards are essential. But it could reduce the time highly trained researchers spend navigating repetitive administrative processes and allow them to devote more attention to the work for which they are uniquely qualified.

Dr Morse’s response to my final question also suggests a possible disconnect between working researchers and the national policy structures intended to support them. The Government has promised national leadership and greater priority for rare cancers, but those commitments will matter only if researchers can see and use them. Should government take a more proactive role in identifying research bottlenecks, connecting laboratories with appropriate funding and ensuring that strategically important projects do not depend entirely on researchers repeatedly having to find and apply to individual funding schemes? How much of the current delay is scientifically unavoidable, and how much is created by systems that could reasonably be improved?

These are questions I intend to raise at the next meeting of the All-Party Parliamentary Group on Brain Tumours in October.

This week’s edition contains five newly announced research, funding and patient-access developments, followed by two spotlights on recruiting clinical trials that deserve wider attention. The distinction matters: the first five items were reported during the week beginning 27 July, while the trial spotlights concern studies that are already under way rather than results newly published this week.

Contents:

1. Australia gets vorasidenib

2. …and announces A$8.4 million for brain cancer research

3. Could earlier focused-ultrasound treatment delay progression of low-grade glioma?

4. A brief postoperative window may help target residual glioblastoma cells

5. Focused-ultrasound delivery into gliomas depends on molecular size

6. Trial Spotlight: GIANT tests checkpoint immunotherapy

7. Trial Spotlight: Antibody-armed T cells combined with focused ultrasound

New developments this week

1. Access Update: Vorasidenib added to Australia’s Pharmaceutical Benefits Scheme

Originator: Agios Pharmaceuticals originally developed vorasidenib; Servier acquired the programme in 2021 and now markets the medicine as Voranigo.

Clinical Sponsor: Servier sponsored the Phase III INDIGO trial on which the access decision is based.

Funder: Servier funded the INDIGO trial.

Campaigning Organisation: Australian Brain Tumour Collaborative and Brain Tumour Alliance Australia were among the organisations that advocated for PBS access.

Reporting Source: Australian Government Department of Health, Disability and Ageing, 2 August 2026; Servier Australia.

Why it matters: This is the week’s clearest immediate benefit for patients. It is not a new research result, but the practical consequence of earlier research: eligible Australians with Grade 2 IDH-mutant astrocytoma or oligodendroglioma can now obtain a targeted oral treatment through the PBS rather than facing a price reported by the government at approximately A$28,000 for a 30-day prescription.

Details

• From 1 August 2026, Voranigo is listed on the PBS for eligible adults and adolescents aged 12 years and over with a susceptible IDH1 or IDH2 mutation, provided they have not previously received systemic anticancer treatment or radiotherapy and meet the remaining PBS criteria.

• The Australian Government estimates that approximately 135 people each year could benefit. Eligible patients will generally pay no more than A$25 per prescription, or A$7.70 with a concession card.

• Vorasidenib inhibits mutant IDH1 and IDH2 enzymes, reducing production of the abnormal metabolite that helps drive these tumours.

• The Phase III INDIGO trial found that vorasidenib prolonged progression-free survival and delayed the need for another intervention in selected patients with residual or recurrent Grade 2 IDH-mutant glioma who had undergone surgery but had not received radiotherapy or chemotherapy.

• The PBS listing should be described as an access and reimbursement development, not as a newly discovered clinical benefit. The evidence supporting it was generated earlier through INDIGO.

• The decision follows sustained submissions and advocacy from patients, clinicians and Australian brain tumour organisations seeking affordable access to the treatment.

Links: Australian Government announcement | INDIGO trial report | Australian Brain Tumour Collaborative submission | Brain Tumour Alliance Australia submission

2. Funding Opportunity: Australia announces A$8.4 million for brain cancer research

Originator: Australian Government Department of Health, Disability and Ageing; Medical Research Future Fund Australian Brain Cancer Mission.

Clinical Sponsor: Not applicable - this is a competitive research grant opportunity, not a clinical trial.

Funder: Medical Research Future Fund, through the Australian Brain Cancer Mission.

Campaigning Organisation: None identified in the announcement.

Reporting Source: Australian Government Department of Health, Disability and Ageing, 30 July 2026.

Why it matters: The announcement makes up to A$8.4 million available for collaborative, translational brain cancer research. It is particularly relevant to the recurring problem of promising laboratory ideas failing to cross the gap into clinical development. The funding is intended to support projects that connect biological understanding, therapeutic strategy and treatment development rather than addressing only one isolated part of the pathway.

Details

• Applicants must propose work that improves understanding in at least two of three areas: factors contributing to brain cancer development; identification and validation of new therapeutic strategies; and development of new therapies.

• The government specifies that projects should be innovative, interdisciplinary, collaborative and translational.

• Eligible work may include preclinical research and early-stage clinical research.

• This is a funding opportunity rather than an announcement of successful projects. No research teams, treatments or trial results have yet emerged from this particular allocation.

• Its eventual value will depend on which projects are selected, how quickly they can begin and whether the grants are large and sustained enough to move credible approaches through recognised research bottlenecks.

Links: Australian Government funding announcement

3. Research Update: Could earlier focused-ultrasound treatment delay progression of low-grade glioma?

Originator: Dr Sophie Morse, Assistant Professor, Department of Bioengineering, Imperial College London.

Clinical Sponsor: Not applicable - this is preclinical research conducted at Imperial College London.

Funder: Astro Brain Tumour Fund.

Campaigning Organisation: Astro Brain Tumour Fund.

Reporting Source: Focused Ultrasound Foundation, distributed through Newswise, 30 July 2026.

Why it matters: This is a direct update on work funded by the Astro Brain Tumour Fund. Its importance lies in treating low-grade glioma as a group of molecularly different diseases and asking whether medicines can be matched to particular subtypes and delivered more effectively by temporarily opening the blood-brain barrier. The long-term aim is not merely to respond after a tumour has become more aggressive, but to delay or prevent that progression where possible.

Details

• Dr Morse’s group is testing a range of drugs with low-intensity focused ultrasound in preclinical models of different Grade 2 glioma subtypes.

• The ultrasound is used with the aim of temporarily opening the blood-brain barrier so that more of the medicine can reach the tumour.

• The work is currently concentrated on two specific subtypes. Subject to further funding, the ambition is to test five drugs across four low-grade glioma subtypes.

• This subtype-by-subtype approach matters because a delivery method will be useful only if the drug being delivered is biologically appropriate for that tumour.

• Low-grade gliomas can progress to higher-grade disease, but the timing and pattern vary greatly between patients. This study is therefore exploring an interception strategy, not claiming that progression can already be prevented.

• Vorasidenib has now added an important treatment option for some eligible patients with IDH-mutant Grade 2 glioma, including through the NHS in England, but further medicines and delivery strategies remain urgently needed.

• The team hopes that lessons from focused-ultrasound studies in high-grade tumours may eventually help shorten the route towards low-grade glioma trials. The present work remains laboratory and animal-model research; no low-grade glioma patient trial has yet been announced from this programme.

• Separately, the Focused Ultrasound Foundation says its UK organisation intends to fund an early clinical trial of sonodynamic therapy for glioblastoma. The article does not identify the trial centre or starting date, so this should be treated as a separate, developing lead.

Links: Focused Ultrasound Foundation report | Astro Brain Tumour Fund | NICE vorasidenib recommendation

4. Research Update: A brief postoperative window may help target residual glioblastoma cells

Originator: Dr Lorena Fernandes and colleagues at the Centre for Nanotechnology in Medicine and Geoffrey Jefferson Brain Research Centre, University of Manchester; collaborating researchers at the Catalan Institute of Nanoscience and Nanotechnology, Barcelona.

Clinical Sponsor: Not applicable - this was a preclinical mouse study.

Funder: Engineering and Physical Sciences Research Council; Rosetrees Trust; Severo Ochoa Centres of Excellence programme.

Campaigning Organisation: None identified for this story.

Reporting Source: Science Translational Medicine; University of Manchester, 29 July 2026.

Why it matters: Glioblastoma generally returns near the edge of the surgical cavity because infiltrating cells remain in brain tissue that cannot safely be removed. This study suggests that surgery itself creates two short periods when circulating liposomal medicines can reach that margin more readily. The attraction is not that standard chemoradiotherapy should immediately be brought forward, but that an additional, precisely timed postoperative treatment might one day be inserted before the usual course begins.

Details

• In mouse models, the researchers identified two periods of blood-brain barrier disruption at the resection margin: immediately after surgery and again approximately 48 to 72 hours later.

• Fluorescently labelled liposomal nanoparticles injected during those periods accumulated preferentially around the surgical margin, while uptake remained low in brain regions where the barrier was intact.

• When the liposomes carried doxorubicin, a single injection during the postoperative window suppressed recurrence and controlled disease across the models tested, with little observed toxicity.

• Liposomal medicines are already used in oncology, which could make translation more straightforward than developing an entirely new drug. However, this exact brain-tumour application, timing and payload are not approved treatments.

• The principle might eventually be extended beyond chemotherapy to carry immunotherapies, RNA medicines or genetic therapies to residual tumour cells.

• The researchers are seeking funding for the next stage. No human trial has yet been announced, so the study should be described as promising preclinical evidence rather than a treatment ready for patients.

Links: Primary paper in Science Translational Medicine | University of Manchester report

5. Research Update: Focused-ultrasound delivery into gliomas depends on molecular size

Originator: Matthew R. Hoch, Victoria R. Breza, G. Wilson Miller and Richard J. Price, University of Virginia.

Clinical Sponsor: Not applicable - this was a preclinical mouse study.

Funder: US National Institutes of Health grants R01EB030409, R01EB030744, R21NS118278 and R01CA226899; University of Virginia Focused Ultrasound Cancer Immunotherapy Center.

Campaigning Organisation: None identified for this story.

Reporting Source: Radiology; UVA Health, 29 July 2026.

Why it matters: Focused ultrasound is a delivery tool, not a treatment in isolation. Its success depends partly on the physical properties of whatever drug, antibody, gene therapy or imaging agent is being carried across the blood-brain barrier. This study provides useful evidence that glioma blood vessels do not necessarily make focused-ultrasound delivery less effective and that there may be an intermediate, “Goldilocks” molecular-size range that produces the best tissue retention.

Details

• The researchers combined focused ultrasound and microbubbles with quantitative susceptibility mapping MRI to measure delivery of contrast agents of different molecular sizes.

• In mouse gliomas, delivery was similar to or greater than delivery into otherwise normal mouse brain, depending on the size of the agent.

• Very small agents were delivered less efficiently than larger ones, while very large agents were also less effective. Intermediate-sized molecules produced the strongest delivery in the experimental system.

• The finding helps answer a practical question for future trials: FUS settings and treatment schedules may need to be matched to the size and behaviour of the intended therapeutic payload.

• The work does not demonstrate that any particular drug improves survival. It is a delivery study designed to make later drug and gene-therapy experiments more rational and measurable.

• The study is especially relevant to the wider FUS field because it suggests that the altered vasculature inside gliomas may remain permissive - and in some circumstances more receptive - to targeted delivery.

Links: Primary Radiology paper | UVA Health report

Clinical trial spotlights

6. Trial Spotlight: GIANT tests checkpoint immunotherapy before glioblastoma surgery

Originator: Dr Mustafa Khasraw and colleagues at Duke University developed the GIANT trial; the biological rationale was strengthened by a foundational case study led by Professor Georgina V. Long and colleagues at Melanoma Institute Australia and the University of Sydney.

Clinical Sponsor: Duke University.

Funder: GIANT trial funding is not separately specified in the accessible public listing. The foundational case study received support from Melanoma Institute Australia, National Health and Medical Research Council Investigator Grants, Cancer Institute NSW, the University of Sydney Medical School Foundation and the CLEARbridge Foundation. Bristol Myers Squibb supplied the checkpoint-inhibitor drugs for the case study but did not design or analyse it.

Campaigning Organisation: None identified for this story.

Reporting Source: Nature Medicine, February 2025; ClinicalTrials.gov and Duke Health trial records, checked 2 August 2026.

Why it matters: Large randomised studies of checkpoint inhibitors have failed to improve survival in glioblastoma. Most tested treatment after the main tumour mass had been removed, while earlier neoadjuvant studies were small and largely involved recurrent disease. GIANT takes a deliberately perioperative approach: stimulate the immune system while the tumour is still present, remove the tumour so that the biological response can be examined directly, and then continue treatment alongside radiotherapy.

Details

• The foundational case involved newly diagnosed IDH-wild-type glioblastoma with an unmethylated MGMT promoter. The patient received one dose of nivolumab, ipilimumab and relatlimab 12 days before maximal safe resection.

• Analysis of the removed tumour found a marked increase in the abundance, diversity and activation of tumour-infiltrating immune cells. Nivolumab was detected bound to T cells within the tumour, showing that the intravenously delivered treatment had reached its intended immune target.

• The Nature Medicine authors were explicit that these biological signs could not establish clinical benefit from a single patient. At the paper’s 17-month data cut-off there was no definitive recurrence, but the patient had also received surgery, radiotherapy and further immunotherapies, so the outcome could not be attributed to the preoperative dose alone.

• The patient and co-author was Professor Richard Scolyer, the internationally recognised melanoma pathologist and researcher. His tumour later recurred, and he died from glioblastoma on 7 June 2026, just over three years after diagnosis. His case generated valuable biological evidence and helped inspire formal testing; it did not prove that the experimental treatment prolonged survival.

• GIANT is an open-label, multicentre, randomised Phase II trial with an initial safety stage. In the safety stage, patients receive nivolumab plus relatlimab before surgery and then the combination with radiotherapy, with or without temozolomide, after surgery.

• In the randomised stage, participants receive either nivolumab alone or nivolumab plus relatlimab before surgery. After resection, all receive nivolumab and relatlimab with radiotherapy; temozolomide is used where appropriate and is omitted in the specified MGMT-unmethylated setting.

• The study is intended for adults with newly diagnosed IDH-wild-type glioblastoma who have not received radiotherapy or chemotherapy. Duke Health lists it as open for enrolment.

• The trial is important because it can test safety, immune activation and clinical outcomes across a defined patient group. Until those results are available, the original case should be treated as a compelling scientific signal rather than evidence of an effective new standard treatment.

Links: Nature Medicine case study | ClinicalTrials.gov: NCT06816927 | Duke Health trial page | Melanoma Institute Australia tribute to Professor Scolyer

7. Trial Spotlight: Antibody-armed T cells combined with focused ultrasound in newly diagnosed glioblastoma

Originator: Dr Camilo Fadul and colleagues at the University of Virginia; the underlying bispecific antibody-armed T-cell platform was developed by Dr Lawrence G. Lum and collaborators.

Clinical Sponsor: University of Virginia.

Funder: Not separately specified in the public registry. Registered trial collaborators are the Focused Ultrasound Foundation and NaviFUS Corporation.

Campaigning Organisation: Focused Ultrasound Foundation, which is also a trial collaborator.

Reporting Source: ClinicalTrials.gov, NCT07343986; Journal of Neuro-Oncology foundational EGFR-BATs study, January 2024.

Why it matters: This Phase I trial addresses one of the central weaknesses of brain-tumour immunotherapy: producing cells capable of attacking cancer is not enough if too few of them enter the tumour. The trial combines targeted immune cells with temporary focused-ultrasound opening of the blood-brain barrier and uses PET imaging to measure whether the cells actually reach the intended tissue.

Details

• BATs means bispecific antibody-armed T cells. A patient’s own T cells are collected, activated and expanded in the laboratory, then coated with an antibody whose two ends recognise CD3 on the T cell and EGFR on a tumour cell.

• The antibody acts as a molecular bridge, directing the T cell towards EGFR-expressing glioblastoma cells. Unlike CAR-T therapy, the cells are not given a permanently inserted genetic receptor; they are armed externally with the bispecific antibody.

• Low-intensity focused ultrasound is applied with circulating microbubbles to open the blood-brain barrier temporarily. The ultrasound is being used for delivery, not to heat or ablate the tumour.

• A particularly strong feature of the design is that some EGFR-BATs are labelled with zirconium-89 oxine (89Zr-oxine). PET scans can then measure trafficking and uptake rather than relying solely on indirect immune markers.

• Arm A receives focused-ultrasound blood-brain barrier opening after the fourth and eighth BAT infusions. Arm B receives it after the first, fourth and eighth infusions. This tests whether beginning ultrasound-assisted delivery earlier changes cell access to the tumour.

• The study is recruiting adults with newly diagnosed, IDH-wild-type, MGMT promoter-unmethylated glioblastoma who meet detailed surgical, performance and tumour-location criteria. Only 12 participants are expected, at a single University of Virginia site.

• The trial builds on an earlier Phase I study of EGFR-BATs without focused ultrasound. Seven patients received the highest feasible total dose of 80 billion armed T cells without dose-limiting toxicity, and three additional patients with MGMT-unmethylated tumours also received weekly infusions without a dose-limiting toxicity. Investigators reported increases in several laboratory measures of anti-tumour immune activity.

• The earlier study reached a manufacturing ceiling rather than a safety ceiling: the team could not reliably expand enough T cells to test the planned higher doses.

• BATs FUS is designed primarily to establish safety, feasibility and proof of delivery. It is far too small to determine whether the treatment extends survival. The registry estimates primary completion in December 2027 and final completion in December 2028.

• This differs from Dr Morse’s low-grade glioma programme. Her work investigates focused-ultrasound-assisted delivery of medicines in preclinical low-grade glioma models; BATs FUS is a clinical trial using focused ultrasound to assist immune-cell delivery in newly diagnosed glioblastoma.

Links: ClinicalTrials.gov: NCT07343986 | Foundational EGFR-BATs study | PubMed record

That’s all for this week.

Note: There’s a lot of articles this week about safusidenib, but we covered that in our NEWSFLASH special edition last Tuesday 21st July - see https://hughmunro.substack.com/p/brain-tumour-news-links-3bc/

Contents of this edition:

1. Nanoparticle + focused ultrasound approach

2. GammaTile brachytherapy implant technology

3. New lab model reveals why immune cells sometimes behave strangely

4. Dordaviprone in diffuse midline glioma

5. Researcher spotlight: Dr Mara De Martino

1. Nanoparticle + focused ultrasound approach shows early promise against glioblastoma (preclinical)

Originator: Roger Abounader, MD, PhD, and team, University of Virginia Comprehensive Cancer Center
Clinical Sponsor: N/A - preclinical, animal studies only, no clinical trial yet
Funder: NIH National Cancer Institute; NIH National Institute of Neurological Disorders and Stroke; UVA Comprehensive Cancer Center; Schiff Foundation; Ben and Catherine Ivy Foundation; Focused Ultrasound Foundation
Campaigning Organisation: None
Reporting Source: UVA Health News, 24 July 2026

Why it matters: This combines two things we track separately - focused ultrasound to open the blood-brain barrier, and a way to hit multiple faulty genes driving glioblastoma at once using microRNA, including genes that currently have no drug at all. Important caveat for readers: this is mouse-model work, not yet tested in people.

Details:

• Brain-penetrating nanoparticles carrying microRNA are guided through the blood-brain barrier using MRI-guided focused ultrasound and microbubbles.

• In mouse models, the approach slowed tumour growth and extended survival, targeting multiple cancer-driving genes simultaneously — including “undruggable” targets.

• Published open-access in the Journal of Clinical Investigation.

Links: UVA Health and Journal of Clinical Investigation

2. GammaTile brachytherapy implant reaches Oregon

Originator/Manufacturer: GT Medical Technologies (Tempe, Arizona)
Clinical Sponsor: N/A - this is clinical adoption of an already FDA-cleared device, not a new trial
Funder: N/A
Campaigning Organisation: None
Reporting Source: OHSU News, 23 July 2026

Why it matters: Not new data - GammaTile’s supporting clinical evidence was presented at ASCO earlier this year - but it’s a real-world access story, showing how far an already-approved technology is spreading and what it offers patients who can’t safely have another course of standard radiotherapy.

Details:

• GammaTile is a biodegradable collagen tile, about the size of a postage stamp, containing radioactive caesium-131 seeds. It is placed directly into the surgical cavity at the time of tumour removal, delivering targeted brachytherapy immediately rather than requiring a separate course of external-beam radiotherapy weeks later. It’s aimed at two groups: patients having repeat surgery after previous radiation, where another course of external beam radiation would be too toxic, and patients having their first surgery, for whom it can replace the radiotherapy normally given about a month later.

• It’s used for both primary brain tumours, including recurrent gliomas, and tumours that have spread to the brain from elsewhere in the body.

• Already FDA-cleared and in use at more than 100 US centres, according to manufacturer GT Medical Technologies. OHSU became the first Oregon hospital to offer it, treating its first patient on 8 July 2026 and two more since.

Link: OHSU News

3. New lab model reveals why immune cells sometimes protect glioblastoma instead of attacking it

Originator: Seokgyu Han, Sun Ha Paek, Sungsu Park and colleagues, Sungkyunkwan University / Seoul National University Hospital, South Korea
Clinical Sponsor: N/A - laboratory/basic science, not a clinical trial
Funder: Not confirmed in the accessible abstract / article metadata
Campaigning Organisation: None
Reporting Source: Neuro-Oncology / Oxford Academic, 21 July 2026

Why it matters: One of the biggest puzzles in glioblastoma is why the immune system so often fails to fight it, even when immunotherapies work well in other cancers. This study built a lab device - a “chip” that lets researchers control the order and timing in which different immune cells meet a glioblastoma tumour - and found that sequence itself changes the outcome. If the brain’s resident immune cells (microglia) reach the tumour first, they tend to switch on suppressive signals that shut down the immune response. If natural killer cells (a different immune cell type that can kill tumour cells directly) arrive first instead, the response stays aggressive. This is an important mechanistic clue, not a treatment - but it does point to a possible way to help chemotherapy work better.

Details:

• The team built triculture models (glioblastoma cells + microglia + natural killer cells) using a “multi-inlet microfluidic platform” that lets them control which immune cell type reaches the tumour first.

• Microglia-first exposure switched on STAT3-driven immunosuppressive signalling; natural-killer-cell-first exposure switched on cytotoxic, interferon-related signalling instead, alongside higher levels of a gene called IL12A - a pattern also seen in more immune-active tumours in real patient data.

• Blocking STAT3 in the lab model reduced the microglia-driven suppression, increased IL12A, and improved the tumour’s response to temozolomide, the standard glioblastoma chemotherapy drug.

• Important caveat for readers: this is a lab-dish model (a “spheroid” chip system), not tested in animals or patients. It’s foundational research explaining why immunotherapy struggles in glioblastoma, several steps removed from anything patients could access.

Link: Neuro-Oncology, Oxford Academic

4. Early rise in a routine MRI measurement predicts who benefits most from dordaviprone in diffuse midline glioma

Originator: Benison C. Lau, Carl Koschmann and colleagues, University of Michigan Medical School and multi-institutional collaborators
Drug developer / licence holder: Dordaviprone / ONC201, marketed as Modeyso by Jazz Pharmaceuticals following FDA accelerated approval
Clinical Sponsor: Not applicable - retrospective multi-institutional imaging-biomarker analysis using patients already treated with dordaviprone
Funder: Not confirmed in the accessible article metadata
Campaigning Organisation: None identified
Reporting Source: Neuro-Oncology / Oxford Academic, 21 July 2026, Open Access

Why it matters: Dordaviprone is the first FDA-approved drug for H3K27M-mutant diffuse midline glioma (DMG) - the tumour category that includes DIPG (diffuse intrinsic pontine glioma) and often affects children and young people. But not everyone responds, and until now doctors have had no early way to tell who’s likely to benefit versus who might need a change of plan sooner. This study found that the standard way of checking tumour size on scans doesn’t predict outcome early on - but a different, already-routinely-collected measurement does.

Details:

• The team analysed 83 dordaviprone-treated patients, narrowing to 38 who received standardised dosing for the main comparison.

• The key measurement is the “apparent diffusion coefficient” (ADC) - a standard part of MRI that reflects how densely packed tumour cells are, without needing any extra scan.

• Patients whose ADC rose after the third treatment cycle (suggesting the tumour was becoming less densely packed with cells) had a median overall survival of 732 days, versus 360 days for those whose ADC fell — roughly two years versus one.

• Progression-free survival showed the same pattern: 334 days versus 110 days.

• Standard tumour-size-based scoring at the same early timepoint did not predict either outcome — only this diffusion measurement did.

• Spinal fluid chemistry testing supported that the ADC rise reflects the drug’s actual biological effect rather than coincidence.

• Important caveat for readers: this is a retrospective analysis identifying a promising signal, not a finished, validated test - the authors themselves say it “warrants prospective validation.”

Link: Neuro-Oncology, Oxford Academic

5. Researcher spotlight: Dr Mara De Martino (Brain Tumour Charity)

Dr De Martino, funded by the Charity’s Future Leaders programme (£225,000 over three years) at Weill Cornell Medicine, found that radiotherapy makes glioblastoma cells change how they use fats - helping them survive treatment and evade the immune system. She’s now moved to UCL’s CRUK Brain Tumour Centre of Excellence to start an independent project asking why glioblastomas recur after treatment.

Reporting Source: The Brain Tumour Charity, 20 July 2026

Link: The Brain Tumour Charity

That’s all for this week

Where the government’s price tag on a year of good health really comes from

Third in a trilogy of special editions, following “The Ten-Year Gap” and “What’s a Year Worth?”

In our recent article ‘What’s a Year Worth?’ (BTNL, July 15th), we used the government’s own price tag for a year of good health - NICE’s (the National Institute for Health and Care Excellence) £25,000 to £35,000 threshold - to work out whether speeding up brain tumour research pays for itself. A fair question for any sceptical reader to ask at that point is: who decides that number, and how? It turns out the honest answer is more interesting, more useful for the argument, and more politically revealing than most people expect.

There isn’t one obvious way to put a price on a year of good health. Government has considered more than one approach - and knowing why one was rejected, and how the one that we use was actually set, matters for judging whether NICE’s £25,000–£35,000 figure is a scientific fact or a policy choice.

The first is the one most people guess: value a life by what that person would have earned, and paid in tax, had they lived. It’s intuitive, and some countries have used versions of it. The UK has largely abandoned it, and for a good reason - it would value a retired person’s life at close to nothing, a disabled person’s less than an able-bodied one, and a stay-at-home parent’s less than, say, a merchant banker’s. No official wants to defend that kind of uncomfortable logic, so this is not what sits behind the figure NICE uses.

The approach that produces NICE’s figure is opportunity cost. Not what a life is worth in any philosophical sense, but a practical constraint - the NHS has a fixed budget, so paying for one expensive treatment necessarily means not paying for something else. NICE’s own guidance says the threshold should reflect ‘the opportunity cost of programmes displaced.’ But the original range was never actually calculated this way. It was fixed in 2004 by committee judgement, not by measurement, at £20,000 - £30,000, and stayed there for two decades. When health economists later tried to estimate the real figure empirically, they found it was probably much lower - somewhere around £13,000 to £18,000. That gap matters: one widely cited estimate suggests the old, over-generous threshold cost the NHS roughly 1.25 million years of good health since 2000, because funding decisions priced above the true opportunity cost necessarily displaced more health elsewhere than they created. From April 2026, the threshold rose to £25,000-£35,000 - the first change since 2004, and, like the original figure, set by ministerial and trade-policy decision rather than a fresh empirical calculation. That’s the figure this article uses throughout: a genuine, current, official number - but one whose history shows it moving by political choice, not scientific discovery.

The two approaches, compared

Why a wobbly number is not a weak argument

A less careful article would cite these figures as settled scientific fact. We’re not going to do that - because the point we’re trying to make is that these thresholds are policy choices, not laws of physics. NICE already applies higher thresholds for severe conditions and highly specialised technologies, and the April 2026 rise to £25,000-£35,000 is itself proof that the ‘official’ figure moves when ministers decide it should. There is no law of nature preventing the threshold from moving again, or from being modified for severity, rarity, unmet need or diseases with unusually poor outcomes. But doing so would be a policy choice, not a scientific discovery - and policy choices are exactly what campaigners and advocates try to influence.

A different, harder kind of logic

Alongside the ‘value of health’ argument, there’s a second, separate case worth making; one closer to what people instinctively mean when they ask whether this is really about tax and public spending. It doesn’t replace the health-value argument from our last substack article; it sits next to it, using different money entirely.

Brain tumours cost the UK an estimated £18.7 billion in total economic burden, for a single year’s new diagnoses. That splits into two genuinely different kinds of cost. £13.5 billion of it is the value of healthy years lost - the same style of calculation as in our article titled ‘What’s a Year Worth?’. But the remaining £5.17 billion is something harder-edged: lost earnings, welfare payments, unpaid care, and direct NHS spending. That is money actually leaving pay packets, the exchequer, and family finances - not an abstract valuation of wellbeing.

So, if annual new diagnosis figures remain broadly steady, each year’s new cases will create another lifetime financial burden of this order. If faster, better brain tumour treatment reduced the lifetime financial burden created by each year’s new diagnoses by even a conservative 5-10% - through earlier access extending working lives, reducing care needs, and cutting NHS costs - the benefit would be worth £258.5m - £517m for each annual cohort of newly diagnosed patients. This benefit would recur cohort by cohort, year after year.

The Covid comparison

Here’s why that recurring figure matters so much. Operation Warp Speed, the US programme that funded and accelerated Covid-19 vaccine development, cost around $13 billion. Independent economic analysis estimated the pandemic was costing the US economy around $26 billion a day. The vaccine programme paid for itself in about twelve hours and went on to save an estimated 140,000 lives and generate roughly $1.8 trillion in economic benefit in its first six months alone. One estimate found the vaccines themselves, sold for around $40 a course, delivered social value of around $5,800 a course - nearly 150 times their price.

The exact figures do not transfer directly - a pandemic and a chronic disease are very different problems. But one part of the logic does transfer: where avoidable delay creates large human and financial costs, public investment in acceleration can be economically rational.

Figure 1: an illustrative annual government investment in research-acceleration infrastructure, set against the value of reducing the lifetime financial burden created by one year’s new UK brain tumour diagnoses.

That ratio - an estimated £12m invested every year, against hundreds of millions of pounds of financial burden reduced for each annual diagnosis cohort - is a smaller multiple than the Warp Speed comparison, but the underlying logic is similar: a modest, sustained government investment set against a recurring cohort-by-cohort benefit that could dwarf it over time. And unlike a hurried illustrative guess, the benefit side of that comparison is built directly from a figure that this Substack has already reported on, with working links; the £12m is Brain Tumour News Links’ illustrative annual funding envelope for the research-acceleration infrastructure. How we arrived at this estimate is set out in full in the notes below.

Putting the whole series together

Three pieces, three separate arguments, all pointing the same way:

• ‘The Ten-Year Gap’ showed that development time can be bought back with money, but with sharply diminishing returns as you approach the biological floor.

• ‘What’s a Year Worth?’ showed that the time you buy back is worth real money, using the government’s own official valuation of a year of health - and that the case holds up even under a cautious reading of it.

• This article, ‘Whose Numbers Are These, Anyway?’ shows those valuations are themselves policy choices rather than fixed truths, and that a second, harder-edged economic case - expressed in pounds and pence - sits alongside them and points the same direction, on a more modest scale than the most successful public health investment of the last decade, but with the same underlying logic.

No single one of these arguments needs to carry the whole case alone. Together they work as a team and are a lot harder to dismiss.

What this doesn’t prove

• The 5–10% reduction in disease burden used above is an illustrative assumption, not a measured result - it hasn’t been tested for brain tumours specifically.

• The £5.17 billion figure covers all UK brain tumours, not one drug or one subtype - it should not simply be added to the vorasidenib-specific figures used in “What’s a Year Worth?”, since that would risk counting some of the same value twice.

• These are estimates built from published figures and established methods, not a fully audited Treasury appraisal. The right next step, if this argument is to be put in front of decision-makers formally, is exactly that kind of audit - not because the underlying logic is shaky, but because the numbers deserve to be pressure-tested by people whose job it is to find holes in them.

The bottom line

The government’s own numbers for the value of a year of life are not scientific constants; they are, even after a headline-grabbing update, still set by committee and ministerial judgement rather than measurement, and openly acknowledged by health economists themselves to be administrative choices, not scientific facts. That’s not a reason to distrust the case for investing in brain tumour research - it’s a reason to make the case twice, from two different directions: firstly using the government’s own official valuations, however imperfect, and then again using hard, recurring, quantifiable costs that never depended on valuing a life at all. Both point the same way. Together, they look a great deal like the argument that got a vaccine programme accelerated in under a year.

Notes on the numbers, for anyone who wants to check them

• NICE’s original £20,000–£30,000 threshold and its origins as a 2004 judgement rather than a calculation, plus later empirical estimates of the true opportunity cost: “Politicised Changes to the NICE Threshold Risk Making Cost-Effectiveness Analysis Performative, Not Informative.”

• Empirical estimates of the NHS’s true marginal cost per QALY (around £13,000–£18,317): Claxton et al., “Methods for the Estimation of the NICE Cost Effectiveness Threshold,” University of York.

• The April 2026 increase to £25,000–£35,000, confirmed as part of the UK-US trade deal on pharmaceuticals: NICE, “Changes to NICE’s cost-effectiveness thresholds confirmed.”

• The £18.7 billion UK brain tumour economic burden figure, split into £5.17 billion direct/fiscal cost and £13.5 billion value of healthy years lost: The Brain Tumour Charity’s “Cost of a Brain Tumour” report.

• Operation Warp Speed cost, daily pandemic cost, lives saved, and economic benefit figures: Institute for Progress, “How To Reuse the Operation Warp Speed Model.”

• The $40-cost/$5,800-social-value estimate per vaccine course: summarised in “The lessons of Operation Warp Speed,” Niskanen Center.

• The £2.8m Tessa Jowell BRAIN-MATRIX investment, initially opened at 10 UK centres: The Brain Tumour Charity, “Tessa Jowell BRAIN MATRIX.”

• The £3.36m 5G platform trial for glioblastoma (Cancer Research UK and Minderoo Foundation, £1.68m each), initially opened at 2 sites: “‘World-first trial’ could change brain cancer treatment,” Brain Tumour Research.

• The national scale (roughly 30 adult neuro-oncology centres; 28 have applied for review) this estimate is scaled against: Tessa Jowell Brain Cancer Mission, “Tessa Jowell Centre of Excellence for Adults.”

How the £12m research-acceleration infrastructure figure was built

This is BTNL’s own conservative estimate, not a published government or charity figure. Scaling the £2.8m BRAIN-MATRIX investment from its initial 10 centres to the ~30-centre national network gives roughly £8.4m (a generous assumption, since a molecular-matching backbone has large, fixed costs that don’t repeat per centre - true marginal cost is likely lower). Adding a conservative allowance to extend 5G-style platform drug-testing capacity beyond its initial 2 sites brings the total to around £12m. The bias throughout is toward overstating rather than understating the true cost.

Editorial note

This article, like the two before it, combines real published figures and established methods into an illustrative model, rather than an audited government appraisal. It’s built to be checked, argued with, and improved - which is surely a more durable foundation for a targeted campaign than just a load of numbers that nobody is allowed to question!

SAFUSIDENIB: THE NEXT STEP AFTER VORASIDENIB?

For years, one question has dominated the thinking of patients, carers and neuro-oncologists alike:

‘What comes after vorasidenib?’

This week (yesterday, 20th July, in fact!), we may have taken the first real step towards answering that question.

Nuvation Bio has announced outstanding long-term results from its Phase 2 study of safusidenib, together with the launch of two major new clinical trials that could reshape the future treatment pathway for people living with IDH1-mutant glioma.

For the first time, researchers are not only attempting to improve on the success of vorasidenib - they are beginning to build what could become a sequence of targeted treatments, each designed to hold the disease at bay for longer.

Remarkable durability

With almost 40 months of follow-up, safusidenib continues to impress.

The updated Phase 2 study reports:

• 52% confirmed objective response rate

• 79% progression-free survival at 36 months

• responses that continue to improve with time, rather than fade.

These are among the most encouraging long-term results yet reported for an investigational targeted therapy for patients who have not undergone chemotherapy or radiotherapy.

The announcement that changes everything

Even more exciting is what comes next:- Nuvation Bio has announced two new clinical studies.

The first is a new international Phase 3 trial in newly diagnosed grade 2 IDH1-mutant glioma, taking safusidenib another major step towards possible regulatory approval.

The second may prove even more important.

Researchers are launching a dedicated study in patients whose grade 2 or grade 3 IDH1-mutant glioma has progressed after treatment with vorasidenib.

This is the question patients have been asking ever since vorasidenib transformed the treatment landscape.

What happens when vorasidenib eventually stops working?

For the first time, a clinical trial is being designed specifically to answer that question.

Building a treatment pathway

Until now, targeted therapy for IDH-mutant glioma has largely meant a single drug.

The future could look very different.

Imagine a treatment journey that begins with surgery, followed by years of disease control using one targeted medicine. If resistance eventually develops, another targeted medicine might take over before radiotherapy or chemotherapy become necessary.

That vision remains unproven, but it is now being actively tested.

If successful, patients could spend many more years living normal lives while postponing treatments that may carry long-term cognitive consequences.

A note of realism

It is important to remember that safusidenib remains an investigational medicine.

It has not yet been approved for glioma, and no study has yet demonstrated that it can successfully control tumours after vorasidenib.

That is precisely what the new trial aims to discover.

Nevertheless, this week’s announcement represents one of the most significant advances in the IDH-mutant glioma field since the arrival of vorasidenib.

Why this is so encouraging

For years, cancer treatment has advanced by replacing one breakthrough with another.

Patients with chronic myeloid leukaemia, melanoma and some forms of lung cancer have all benefited from successive generations of targeted therapies that steadily extended survival.

Could IDH-mutant glioma now be beginning the same journey?

It is far too early to know.

But for the first time, there is genuine reason to believe that vorasidenib may not be the end of the story - it may simply be the beginning.

We’ll be following the development of safusidenib closely and reporting every significant milestone as it happens.

Originator: Nuvation Bio Inc. (NYSE: NUVB)
Clinical Sponsor: Nuvation Bio Inc.
Collaborating Institutions: Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine
Funder: Nuvation Bio Inc.
Campaigning Organisation: American Brain Tumor Association (supporting statement)
Reporting Source: Nuvation Bio press release, 20 July 2026

Links: Nuvation Bio: full press release, 20 July 2026 ; ClinicalTrials.gov: existing SIGMA trial (NCT05303519)

In this edition we have:

1. A live UK focused-ultrasound trial

2. The ‘super sponge’ - a new approach to bypassing the blood brain barrier

3. EGFR inhibitors may make TMZ more effective

4. Gamma-delta T cells encouragingly effective against glioblastoma in mouse models

5. Assessing patient suitability for low grade IDH-mutant glioma treatments

6. Quality of life for adults with low grade tumours

As always, early-stage research should be treated with caution. Preclinical results are not patient results, and Phase I trials are designed mainly to test safety and feasibility, not to prove that a treatment works.

1. Trial Alert: Sonodynamic Therapy Trial Recruiting at St Mary’s, London

Originator: Prof Wladyslaw Gedroyc, Imperial College Healthcare NHS Trust
Clinical Sponsor: Imperial College Healthcare NHS Trust
Funder: Focused Ultrasound Foundation
Campaigning Organisation: None identified for this story
Reporting Source: NIHR Be Part of Research; ISRCTN registry entry

Why it matters

This is a live, currently recruiting UK trial using focused ultrasound in glioblastoma patients.

It is different from the blood-brain-barrier opening work often discussed in relation to focused ultrasound. This trial is testing sonodynamic therapy: using focused ultrasound to activate a tumour-localising compound already used in glioblastoma surgery.

The trial is early-stage - Phase I, safety and feasibility focused - so it is not a proven treatment. But this is genuinely actionable information for anyone in the eligible population, because it is recruiting at a named UK hospital with a formal trial record.
It is one to watch closely. It is early, risky and unproven, but it is also real, regulated, UK-based and recruiting. For readers, the important point is that focused ultrasound is no longer just a laboratory idea in brain tumours; at least some versions of the technology are now being tested in patients.

Details

• The trial combines 5-ALA - 5-aminolevulinic acid - with MRI-guided, low-frequency focused ultrasound.

• 5-ALA is already used in high-grade glioma surgery because it accumulates in tumour tissue and helps surgeons see tumour cells under blue light.

• In this trial, ultrasound is intended to activate 5-ALA within the targeted tumour tissue, producing local cytotoxic effects. This is similar in principle to photodynamic therapy, but uses sound rather than light so the energy can be focused through the skull.

• Patients receive the sonodynamic therapy procedure about a week before planned surgery.

• MRI is used immediately afterwards and again at follow-up before surgery.

• The treated tissue can then be assessed after surgery for signs of biological effect.

• Eligible participants are adults aged 18–70 with suspected primary lobar glioblastoma.

• There is a substantial list of exclusion criteria, including bleeding risk, certain metal implants, cardiac conditions, MRI incompatibility and other safety considerations.

• The trial record states that early evidence shows no significant side effects in five patients, but this should be treated only as an early safety signal, not evidence of efficacy.

• Recruitment is open at Imperial College Healthcare NHS Trust, St Mary’s Hospital, London, from February to November 2026.

Links: NIHR Be Part of Research: full trial details | ISRCTN registry entry

2. Research Spotlight: The “Super Sponge” - a different way to tackle glioblastoma’s blood-brain-barrier problem

Originator: Dr Ben Newland, School of Pharmacy and Pharmaceutical Sciences, Cardiff University
Collaborating Institutions: Universities of Nottingham, Birmingham, Sheffield, and King’s College London
Clinical Sponsor: Not applicable - translational/preclinical research; not yet in clinical development
Funder: Brain Tumour Research, in partnership with the Medical Research Council. The project received an original £500,000 award, linked to a wider £2 million government investment announced in 2023, plus a further £75,000 extension announced this month.
Campaigning Organisation: Brain Tumour Research
Reporting Source: Cardiff University; Brain Tumour Research

Why it matters

Many recent drug-delivery stories in this newsletter have focused on ways to get treatments through the blood-brain barrier - focused ultrasound, nanoparticles, carrier systems and similar approaches.

Dr Ben Newland’s “Super Sponge” project takes a different route. It aims to bypass the blood-brain barrier by placing the drug source directly into the surgical cavity left after glioblastoma removal. That matters because recurrence commonly begins near the original tumour site, where residual infiltrating tumour cells remain after surgery.

This month’s update brings concrete progress. The sponge has now been shown safe for long-term implantation in mice, and laboratory testing shows it can release drugs over many months.

Details

• The “Super Sponge” is a soft, highly porous, compressible material called a cryogel.

• It is designed to be loaded with cancer-fighting drugs and implanted directly into the surgical cavity after tumour removal.

• The aim is to target both the blood-brain-barrier problem and the residual tumour cells that drive recurrence.

• The team is screening thousands of existing, already licensed drugs - repurposed rather than newly invented - to find those most harmful to glioblastoma cells while sparing healthy brain tissue.

• A 2024 conference abstract showed progress to 3D-printed, well-defined cylindrical cryogels tested with candidate drugs including clemastine, an existing antihistamine.

• New this month: the sponge has been confirmed safe for long-term implantation in mice, and laboratory testing shows it can release drugs over many months.

• The next step is loading the sponges with selected drugs and testing efficacy in animal models.

• The £75,000 extension will fund continued drug screening.

• Dr Newland has been candid about the difficulty ahead: “The jump from lab to clinical trial is really hard, but I am determined to try and make a difference for patients.”

This is not close to routine clinical use, but it is aimed at turning drug-delivery theory into something surgeons might one day actually use.

Links: Cardiff University: original announcement | Brain Tumour Research: funding boost and full interview | Brain Tumour Research: Senedd showcase update

3. EGFR inhibitors may make glioblastoma more sensitive to temozolomide

Originator: Dr Amyn Habib, Professor of Neurology and Neurological Surgery, UT Southwestern Medical Center; Staff Physician, Dallas VA Medical Center
Collaborating Institution: University of Alabama at Birmingham
Clinical Sponsor: Not applicable - preclinical research, not a glioblastoma clinical trial
Research Institutions: UT Southwestern Medical Center; University of Alabama at Birmingham
Funder: National Institutes of Health; National Cancer Institute Cancer Center Support Grant
Campaigning Organisation: None identified for this story
Reporting Source: UT Southwestern Medical Center Newsroom, 8 July 2026, reporting on Science Translational Medicine

Why it matters

This is a preclinical study proposing a practical strategy: rather than developing an entirely new glioblastoma drug, it asks whether existing EGFR inhibitors - drugs already approved in other cancers, but not approved for glioblastoma - could make temozolomide work better.

Temozolomide remains the standard chemotherapy used in glioblastoma, but tumours often become resistant to it. If that resistance could be reduced or reversed, even partly, it could matter.

This is now the second chemosensitisation story in two weeks. That pattern is worth noting. It suggests a broader shift in the field towards “make TMZ work better” rather than only “replace TMZ”. Repurposing or combining with existing approved drugs may offer a shorter route to patients than developing something entirely new.

This is really worth watching because it uses already-known drugs to tackle a known resistance problem. But it remains preclinical. The question now is whether the strategy can be translated safely and effectively into human glioblastoma trials.

Details

• Researchers found that treating glioblastoma models with EGFR inhibitors made tumours more sensitive to temozolomide.

• The study looked at a mechanism involving MGMT, a DNA-repair protein associated with resistance to temozolomide.

• The findings are from preclinical models and have not yet been tested in glioblastoma patients.

• Dr Habib suggested that, if future clinical trials confirm the effect, this kind of combination could eventually become important in treatment. That is the investigator’s forward-looking view, not a clinical conclusion yet.

• The study was co-led with the University of Alabama at Birmingham.

NB:
This study was published on 8 July, just before the window for this edition. I am including it here because it fits with the emerging chemosensitisation theme and was not covered last week.

Links: UT Southwestern Medical Center Newsroom

4. Gamma-delta T cells eliminate glioblastoma in mouse models, company reports

Originator: Investigators at Taipei Medical University, in collaboration with JY BioMed and HeXun Biosciences
Developer / Company Sponsor: SL Science Holding Limited
Clinical Sponsor: Not applicable - preclinical programme; no human trial reported for this specific product yet
Funder: Not specified in available sources
Campaigning Organisation: None identified for this story
Reporting Source: GlobeNewswire / SL Science press release, 17 July 2026; presentation at the 20th World Congress of Basic and Clinical Pharmacology, Melbourne

Why it matters

This is preclinical, company-reported data - not yet peer-reviewed or published - so it needs the usual caution around company press releases.

That said, the result reported is striking: complete tumour elimination in mouse models at the highest tested dose. The approach also has a different rationale from many T-cell therapies previously covered here.

Gamma-delta T cells are a less common class of T cells. One of the reasons they are interesting in cancer is that they may recognise tumour cells without relying on the usual MHC presentation system - a system glioblastoma can exploit or disrupt as part of immune escape.

That is a real theoretical advantage. But the important question is whether it survives the jump from mice to humans, where glioblastoma immunotherapy has a long history of promising preclinical data failing to translate.

Details

• The therapy uses repeated direct-to-brain / intracranial dosing of expanded gamma-delta T cells in preclinical glioblastoma models.

• Results were dose-dependent.

• Higher ratios of immune cells to cancer cells produced stronger tumour control.

• Complete tumour eradication was reported at the highest tested dose, an 8:1 immune-cell-to-cancer-cell ratio, by day 26 post-treatment.

• Separately, SL Science announced on 10 July 2026 that it had submitted an orphan-drug designation request to the US FDA for its Vdelta2+ gamma-delta T-cell product for glioblastoma. The company’s designation details list FDA receipt as 30 March 2026, and the request remains under review.

• These are mouse-model results only. No human trials have been reported for this specific programme yet.

Links: BioSpace: WCP 2026 presentation announcement | BioSpace: FDA orphan-drug designation request, 10 July 2026

5. Modelling reveals three distinct ways low-grade IDH-mutant glioma responds to chemotherapy

Originator: Dr Amélie Darlix and colleagues, Montpellier Cancer Institute and Montpellier University Hospital, France
Clinical Sponsor: Not applicable - retrospective modelling study, not a clinical trial
Research Institution: Montpellier Cancer Institute / University of Montpellier
Funder: Not specified in available sources
Campaigning Organisation: None identified for this story
Reporting Source: Neuro-Oncology Advances, published 13 July 2026, open access

Why it matters

This sits well alongside the Weill Cornell methylation story covered previously. Both are about the same underlying puzzle: why do IDH-mutant gliomas, often described as one broad disease category, behave so differently from patient to patient?

Rather than presenting a new treatment, this paper offers a new way of understanding who chemotherapy may actually help.

Finding that the majority of patients had durable, long-lasting responses is genuinely encouraging. But identifying that a meaningful minority either do not respond at all, or respond only temporarily before rapid regrowth, is just as important.

That kind of work could eventually help clinicians identify earlier which patients need a different strategy, rather than waiting months or years to see whether standard treatment is working; it could eventually help move treatment away from “try it and wait” towards earlier prediction of who is genuinely benefiting.

Details

• Researchers analysed 1,956 MRI scans from 195 patients with lower-grade IDH-mutant glioma treated with first-line chemotherapy at a single French institution.

• They used an automated AI segmentation tool to track tumour size over time.

• Statistical modelling identified three response patterns:

  • Non-response group: 14% of patients, with early tumour growth and median overall survival of 41 months.

  • Durable-response group: 57% of patients, with stable or shrinking tumours and median overall survival of 169 months.

  • Transient-response group: 29% of patients, whose tumours initially shrank before regrowing rapidly within about two years, with median overall survival of 54 months.

• The authors argue that these patterns are strongly linked to progression and malignant transformation.

• They also highlight the need for early biomarkers to identify which response category a patient is likely to fall into.

Links: Neuro-Oncology Advances: full paper

6. Three new grants target quality of life for adults with low-grade brain tumours

Originator: Mr Will Bolton, University of Leeds; Sarah Rimmer and Jenna Sendall, Cambridge University Hospitals NHS Foundation Trust; Dr Florien Boele, University of Leeds
Clinical Sponsor: Not applicable; quality-of-life research awards, not treatment trials
Research Hosts: University of Leeds; Cambridge University Hospitals NHS Foundation Trust
Funder: The Brain Tumour Charity Quality of Life Award - up to £100,000 per project, approximately £300,000 total
Campaigning Organisation: The Brain Tumour Charity
Reporting Source: The Brain Tumour Charity, 16 July 2026

Why it matters

This is a different kind of research story from most of what is covered here. It is not a drug, device or delivery mechanism. It is about the long-tail, everyday impact of living after a low-grade brain tumour diagnosis.

That matters because progress cannot only mean longer survival. It must also mean better support, better function, better relationships, better return to work, and a better life after treatment.

It is also a good example of research shaped directly by patients. The projects came out of a “sandpit” event where community voice representatives with lived experience sat alongside researchers and clinicians to identify important gaps in support.

Details

• Understanding changing needs - Will Bolton, University of Leeds: developing a way to check patients’ support needs more regularly than fixed clinic appointments allow, aiming to catch problems earlier and reduce anxiety.

• Returning to work - Sarah Rimmer and Jenna Sendall, Cambridge University Hospitals NHS Foundation Trust: identifying barriers adults face when returning to work after diagnosis and treatment, to inform tailored support programmes.

• Intimacy and relationships - Dr Florien Boele, University of Leeds: researching how low-grade brain tumours affect intimacy, relationships and self-image, an area too often left out of clinical conversations.

• The sandpit event that generated these projects took place in Birmingham in November 2025.

• The Brain Tumour Charity also highlighted the case of Callum Blackwell, 24, diagnosed with craniopharyngioma in 2025, who described fatigue, brain fog and memory problems that have affected his ability to work.

This is patient-centred research. If people are living longer with low-grade brain tumours, the research agenda has to include how they live - not just how long.

Link: The Brain Tumour Charity: full announcement

That’s all for this week. If anyone has any suggestions on how improve this substack - please shout at me!

Weighing the true value of speeding up brain tumour research

If you have ever paid someone to finish a job faster than usual, you will know the pattern. The first bit of extra speed is often affordable. The last bit is expensive. At some point, you are no longer paying for ordinary work. You are paying for more people, more equipment, more parallel activity, and more risk being taken up front.

Medical research works in a similar way.

Before a new drug reaches a patient, it must pass through discovery, laboratory testing, safety studies, clinical trials, regulatory review and health-system adoption. Some of that time can be compressed; more researchers can be put on the problem, more compounds can be screened at once, more laboratories can run experiments in parallel. Manufacturing can sometimes begin before the final answer is known.

But some of the waiting is real. In safety testing, for example, researchers may need to observe what happens in the laboratory over weeks or months. You cannot observe a 90-day safety signal in 30 days, no matter how urgent the cause or how generous the funding.

That’s why speed gets expensive. The first months saved may be bought efficiently. The last months become harder, because you are no longer fighting only bureaucracy or lack of capacity. You’re fighting biology itself.

But that’s only half the picture.

The other half is this: what is a year of earlier access worth?

For families living with a brain tumour, the answer is obvious in human terms. A year can mean another Christmas. Another birthday. Another family holiday. Another scan that says stable. Another year before radiotherapy, chemotherapy or surgery has to begin. Another year of work, independence, parenting, studying, campaigning, living.

But public bodies also put numbers on these things. They have to. When the NHS decides whether to fund a new treatment, or when government departments judge whether a public-health policy is worth the cost, they need a common yardstick. It sounds cold, but without a yardstick there is no consistent way to compare one intervention with another.

The usual NHS measure is the quality-adjusted life year, or QALY. In plain English, one QALY is one year of life in full health, or a longer period in less than full health, adjusted for quality of life. NICE’s current standard threshold is around £25,000 to £35,000 per QALY. In wider government appraisal, the Green Book approach used by government departments can use a higher value, around £70,000 per QALY.

Those numbers are not perfect. They are not moral truths. They do not capture grief, fear, disability, exhaustion, family trauma, or the sheer brutality of a brain tumour diagnosis. But they are official numbers. They are the numbers the system itself uses when deciding whether something is “worth it”.

So, let’s use them.

A real example: vorasidenib

To make the argument concrete, take one real brain tumour drug: vorasidenib.

Vorasidenib is not a treatment for classic IDH-wildtype glioblastoma. It is a targeted drug for a different group: people with Grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery. These are generally slower-growing tumours, but they are still serious, life-changing and incurable diseases for many patients.

In the INDIGO trial, patients receiving vorasidenib had a median progression-free survival of 27.7 months, compared with 11.1 months for those receiving placebo. In other words, the drug delayed tumour progression by about 16.6 months on average. (N.B. That was the original trial on which access was based – since then vorasidenib has shown continued performance improvement.)

Progression-free survival is not exactly the same as a quality-adjusted life year. It should not be pretended that it is. But in this particular case, progression-free time is a meaningful measure because the treatment is designed to delay tumour growth and postpone the need for more aggressive interventions such as radiotherapy and chemotherapy. Later follow-up has also supported the idea that patients can remain well on treatment, with health-related quality of life and neurocognition preserved.

Now ask a simple question. What if a drug like this had reached patients one year earlier?

A conservative England-only estimate is that an annual cohort of around 300 people may benefit. A broader UK scenario might use around 400 patients. By accelerating access by one year, we unlock the full clinical value of the treatment for an entire annual cohort of patients who would otherwise face progression during that year of delay. Each patient gains, on average, around 1.38 years before tumour progression.

Using the lower end of the NICE threshold, £25,000 per QALY:

• 300 patients × 1.38 years × £25,000 = £10.35 million

• 400 patients × 1.38 years × £25,000 = £13.8 million

Using the wider Green Book government appraisal value of around £70,000 per QALY:

• 300 patients × 1.38 years × £70,000 = £28.98 million

• 400 patients × 1.38 years × £70,000 = £38.64 million

So even on a cautious calculation, one year of earlier access to one brain tumour drug, for one specific patient group, could represent roughly £10 million to £39 million of health value in England or the wider UK, depending on which official valuation is used.

That is not a miracle claim. It is not saying every pound automatically produces a cure. It is not saying progression-free survival is identical to perfect health. It is simply saying this:

Delay has a value. Speed has a value. And the value of speed can be measured.

Does speeding up pay for itself?

Figure 1: The graph below puts the two sides of the argument together. Worked example: one brain tumour drug, using a 400-patient UK scenario. The cost curve is an illustrative project-crashing model (a project management framework mapping the escalating costs of accelerating a timeline); the value lines use official health-valuation methods. This is not a formal economic appraisal, but a transparent way of showing the value of time.

The rising curve shows the extra cost of trying to make the research and access pathway faster. It starts relatively gently, because early gains can often be bought by removing bottlenecks, funding more capacity, and running work in parallel. But then it climbs more steeply, because the closer we push towards the fastest possible timeline, the more expensive each additional month becomes.

The two straight lines show the value of earlier access, using one real brain tumour drug as a worked example.

The cautious line uses the midpoint of NICE’s usual cost-effectiveness range: £30,000 for one quality-adjusted year of life.

The higher line uses the wider Green Book value used in government appraisal: around £70,000 for one quality-adjusted year of life.

The calculation is deliberately conservative. It uses a 400-patient UK working scenario and the INDIGO trial’s original median progression-free survival difference of about 16.6 months.

On the NICE midpoint valuation, the model breaks even at around 1.2 years of acceleration. In plain English, if targeted funding could bring a drug like this to patients just over a year earlier, the health value gained would broadly match the extra cost in this illustrative model.

On the wider Green Book valuation, the case remains positive until roughly 2.8 years of acceleration.

That is the important point. The argument does not require fantasy numbers. Even on cautious assumptions, a modest acceleration can pay for itself in health value. A more ambitious acceleration needs the broader government valuation to justify it, but that is still an official valuation, not a number invented for a campaign.

What the calculation does - and does not - prove

This model does not prove that any particular injection of cash will produce a specific number of extra life-years. It does not prove that £1 million spent today will bring a drug to patients exactly six months sooner. It does not prove that all drug-development timelines can be compressed equally.

What it does show is more modest, but still powerful: when a treatment works, delay is not neutral. Every year lost before access has a measurable human and economic cost.

For brain tumour research, that matters enormously. The usual public discussion focuses on how expensive research is. That is true. Research is expensive. Clinical trials are expensive. Drug development is expensive.

But delay is expensive too.

The cost of going slowly is usually hidden because no invoice arrives. Families pay it in uncertainty, progression, disability, lost independence, lost work, lost time and grief.

That hidden cost can be made visible.

Where targeted funding can buy time

The argument is not that a lump of money alone can buy an entire drug-development programme. It cannot. The decision-making processes in medical research are far too complex for that to be the case.

The argument is that there are sequences within these development programmes that can be compressed and that these junctures and links within the system provide opportunities in which money really can buy time.

Examples are:

• faster molecular diagnosis, so patients are matched to the right treatment or trial sooner

• better trial-matching systems, so patients do not lose months trying to find options

• preclinical screening capacity, including organoid and patient-derived tumour models

• drug-delivery research, including work on the blood-brain barrier

• early-career fellowships for scientists committed to brain tumour research

• data-sharing infrastructure, so results can be reused instead of rediscovered

• seed funding for high-risk ideas that are too early for industry but too promising to ignore

These are not abstract ambitions. They are practical bottlenecks. They are the places where more money, well directed, can significantly reduce waiting time.

In project management, this is sometimes called “crashing” a project: spending more to shorten the timeline by running work in parallel, adding capacity, or removing bottlenecks. It does not remove the biological floor. It does not make safety testing optional. But it can certainly remove avoidable waiting.

That distinction matters. We are not claiming that money can defeat biology. But we are claiming that money can reduce avoidable and costly delays that biology does not require.

What this does not take into account

In the interests of not overstating the case, this calculation leaves out a great deal.

It works through one drug, for one type of brain tumour. The total value of speeding up the wider brain tumour research pipeline would be larger, because it would include many tumour types and many possible interventions.

It counts only the delay before tumour progression, not the full value of delaying radiotherapy, chemotherapy, cognitive decline, seizures, loss of work, loss of independence, or the emotional burden on families and carers.

It does not put a price on parents having more time with their children, partners having more time together, or young adults being able to continue building their lives.

It does not include the wider economic value of people remaining in work or education for longer.

It does not include the knowledge gained from faster trials, even when a treatment fails. Negative trials can still save future patients from ineffective treatment and redirect research money faster.

So, the numbers here are not inflated. If anything, they are deliberately narrow.

Why this matters for financial decision makers

Policymakers, major donors, foundations and corporate supporters need to know what the money is for, why it matters, and whether the argument survives scrutiny.

This is where the value-of-speed argument helps.

It says:

• brain tumour research is not only underfunded; it is slowed by identifiable bottlenecks

• delay has a measurable cost

• earlier access to an effective treatment has measurable health value

• targeted funding can reduce avoidable delay

• even cautious official valuation methods support the case for acceleration

That is a different pitch from simply saying, “Brain tumour research needs more money.”

It says, “Here is what delay costs. Here is where money can buy time. Here is why buying time is worth it.”

The bottom line

For patients and families, faster research is not an abstract policy goal. It is the difference between waiting and access. Between watching and acting. Between being told that something promising exists somewhere in the pipeline and actually having a chance to receive it.

For financial decision makers, the message is equally clear. Funding brain tumour research is not just an act of compassion. It is an investment in time - and time has measurable value.

And that is why speeding up brain tumour research is a fundraising case that can stand on both moral and economic grounds.

Notes on the numbers

• NICE’s current standard cost-effectiveness threshold is around £25,000 to £35,000 per quality-adjusted life year — this reflects the change that took effect in April 2026, raised from the previous £20,000–£30,000 range used for the prior 20 years.

• Wider government appraisal may use a higher Green Book value, around £70,000 per QALY.

• The vorasidenib trial example is based on the INDIGO trial, which reported median progression-free survival of 27.7 months with vorasidenib and 11.1 months with placebo.

• Vorasidenib is approved for Grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, not for classic IDH-wildtype glioblastoma.

• The 300-patient England estimate is not an invented assumption: it is The Brain Tumour Charity’s own published figure for people in England eligible for vorasidenib, independently corroborated by Brainstrust. The broader 400-patient UK-wide scenario is our own extrapolation from that figure, scaled up for Scotland, Wales and Northern Ireland, and should be read as an estimate rather than an official count.

• The graph uses a simplified project-crashing cost curve to show the likely shape of acceleration costs. It is illustrative, not a formal audited model.

• This piece combines established health-economic concepts with published trial and access figures. It is designed to be transparent and checkable, not to replace a formal government or academic economic appraisal.

Sources and further reading

• NICE cost-effectiveness threshold guidance

• HM Treasury Green Book appraisal guidance

• FDA vorasidenib approval announcement

• Mellinghoff et al., INDIGO trial, New England Journal of Medicine

• Brain Tumour Charity report on vorasidenib NHS approval

• RAND Europe / Brunel University / King’s College London work on the economic returns from UK cancer research

• Value in Health research on the cost of delayed cancer treatment access

1. Restoring chemotherapy sensitivity in resistance glioblastoma

2. A patient’s personal account of the NHS access gap

3. Progression-free survival doubled by repeated injections

4. Why IDH-inhibitors don’t work for everyone

5. Sugar-coated nanoparticles help to get across the blood brain barrier

6. Rare Cancers Bill - implemention and the role of Brain Cancer Justice

1. Experimental compound may restore chemotherapy sensitivity in resistant glioblastoma

Originator / Senior Author: Prof Haitham Amal, Hebrew University of Jerusalem
Collaborating Institution: Harvard Medical School
Clinical Sponsor: Not yet in clinical development - preclinical stage
Funder: Not specified in available sources
Campaigning Organisation: None identified for this story
Reporting Source: GlobeNewswire, 9 July 2026 / Bioengineer.org, 10 July 2026, reporting on Cancer Medicine (embargoed publication date 15 July 2026)

Why it matters: Temozolomide resistance is one of the biggest practical obstacles in glioblastoma treatment - many tumours simply stop responding to the one chemotherapy drug that’s been standard-of-care for over 25 years. This study takes a “re-sensitising” approach rather than trying to replace TMZ outright, which is a genuinely different strategy worth watching, though it’s still very early (preclinical models only, no human data yet).

Details:

• Researchers identified nitrosative stress - an overproduction of nitric oxide that helps tumour cells survive and resist treatment - as a target for re-sensitising glioblastoma to chemotherapy.

• The experimental compound BA-101 selectively inhibits neuronal nitric oxide synthase (nNOS), the enzyme driving this process.

• In preclinical models, combining BA-101 with temozolomide substantially slowed tumour growth, reduced invasiveness, and induced cancer cell death more effectively than either drug alone.

• The compound has been licensed to biotech company NeuroNOS (co-founded by Prof Amal) for further development.

• The team is explicit that extensive preclinical validation and clinical trials are still needed before this could reach patients.

Links: GlobeNewswire report, 9 July 2026 | Bioengineer.org coverage, 10 July 2026

2. Patient voice: “The system doesn’t support it” - a personal account of the NHS access gap

Originator: Hannah Lemanski, brain tumour patient and Involvement Champion
Campaigning Organisation: The Brain Tumour Charity
Reporting Source: The Brain Tumour Charity, 9 July 2026

Why it matters: This is a first-person companion piece to the Charity’s Unlocking Innovation for Brain Tumours in the UK report (which you may recall from earlier BNOS coverage), and it puts a human face directly on exactly the “science moves faster than the system” tension we’ve been talking about. It’s also got some striking on-record quotes from policymakers.

Details:

• Hannah, diagnosed with a central neurocytoma, was refused NHS funding for stereotactic (gamma knife) radiotherapy for her specific tumour type despite her consultant recommending it — she ultimately self-funded treatment costing tens of thousands of pounds.

• She represented the patient voice at a Westminster roundtable (September 2025) and the report’s launch panel (March 2026), alongside The Brain Tumour Charity’s Chief Executive Dr Michele Afif, Prof Mike Lewis (University of Birmingham), George Freeman MP, and Dr Amit Aggarwal (ABPI).

• Dr Aggarwal’s assessment: the UK has “an exceptional science base... where we struggle is in that translation into clinical benefit” - no single part of the system fails, but collectively it isn’t optimised end-to-end.

• George Freeman MP noted that roughly 40 separate decisions currently need approval along the supply chain to change a treatment pathway, and floated an “opt-out” model for clinical trial enrolment (like organ donation) using an app-based consent system.

• Brain tumour trials have the lowest patient recruitment rate of any cancer type.

Links: Read Hannah’s full account | Unlocking Innovation for Brain Tumours in the UK report

3. Repeated injections double progression-free survival in glioblastoma

Originator / Principal Investigator: Dr. Louis B. (”Burt”) Nabors, Professor of Neurology, Director of Neuro-Oncology, O’Neal Comprehensive Cancer Center, University of Alabama at Birmingham
Clinical Sponsor: University of Alabama at Birmingham Neuro-Oncology Program
Funder / Industry Partner: IN8bio, Inc. (developer of the DeltEx DRI platform; UAB intellectual property licensed to the company)
Campaigning Organisation: None identified for this story
Reporting Source: CURE Today, 9 July 2026, reporting on the Journal of Clinical Oncology

Why it matters: This is a genuinely different angle on cellular immunotherapy for glioblastoma - instead of engineering cells to recognise a specific tumour antigen (as with the CAR-T stories covered in this substack recently), this approach makes the patient’s own gamma-delta T cells chemotherapy-resistant, so they can be delivered repeatedly through a catheter left in the tumour cavity, right alongside ongoing temozolomide treatment. The dose-response relationship - more injections correlating with meaningfully longer progression-free survival - is the most interesting signal here, and the safety profile (no dose-limiting toxicities, no cytokine release syndrome across any cohort) is genuinely reassuring for a first-in-human study. As ever with a 13-patient Phase I trial, this needs a larger study to confirm, but it’s a clean, well-conducted piece of work.

Details:

• The therapy (DeltEx DRI) uses the patient’s own gamma-delta T cells, genetically modified to express MGMT so they survive alongside temozolomide, then injected directly into the tumour cavity via a Rickham catheter placed during surgery.

• Patients who received repeated doses (up to 6, across escalating cohorts) had a median progression-free survival of 16.1 months, versus 8 months for those who received a single dose.

• Median overall survival was 19.5 months for repeat-dose patients versus 15.6 months overall, with some patients remaining alive and progression-free at data cutoff (up to 51 months in one case).

• No dose-limiting toxicities, cytokine release syndrome, or neurotoxicity (ICANS) were observed in any of the three cohorts.

• Of 23 patients assessed for eligibility, 13 received the full therapy; there were no treatment-related deaths.

• Trial registration: NCT04165941. Journal reference: Nabors LB, et al., “Intracranial Injection of Investigational Ex Vivo Expanded and Activated Gamma-Delta T Cells Engineered With a Methylguanine-DNA Methyltransferase–Expressing Lentivector in Patients With Primary Glioblastoma,” Journal of Clinical Oncology (2026).

Links: CURE Today report | UAB News release | ClinicalTrials.gov: NCT04165941

4. Cell-by-cell mapping reveals why IDH-mutant gliomas turn aggressive - and why the IDH-inhibitor drug doesn’t work for everyone

Originator / Co-Senior Authors: Dr. Dan Landau, Weill Cornell Medicine, New York Genome Center; Dr. Mario Suvà, Massachusetts General Hospital / Mass General Brigham Cancer Institute
Co-First Authors: Drs. Masashi Nomura, Ramya Raviram, Joshua S. Schiffman
Clinical Sponsor: Weill Cornell Medicine, New York Genome Center, Harvard Medical School, Mass General Brigham
Funder: National Cancer Institute and National Human Genome Research Institute (NIH); STARR Cancer Consortium; The Mark Foundation Emerging Leader Award; The Sontag Foundation; MacMillan Family Foundation
Campaigning Organisation: None identified for this story
Reporting Source: Weill Cornell Medicine Newsroom, 10 July 2026, reporting on Nature Genetics (published 22 June 2026)

Why it matters: This is a mechanism story rather than a trial result, but it needs to be told because it’s about vorasidenib. It offers a biological explanation for something we’ve reported on before in this substack, without fully being able to explain: why an IDH-inhibitor drug only benefits a subset of IDH-mutant glioma patients. If the researchers’ hypothesis holds up, it points toward a future biomarker - a way to identify in advance which patients are likely to respond to IDH-inhibitor treatment and which are not, rather than the current one-size-fits-all approach.

Details:

• Researchers applied advanced single-cell profiling techniques (rather than traditional bulk tissue analysis) to 36 tumour samples taken at different time points and grades from the same IDH-glioma patients.

• IDH gliomas typically start as slow-growing tumours with high levels of gene-silencing DNA methylation, but become faster-growing and more aggressive as they progressively lose those methylation marks.

• The study found this progressive hypomethylation is linked to glioma cells shifting into immature, stem-cell-like states that are more treatment-resistant and better able to spread within brain tissue.

• The team’s working hypothesis: patients whose IDH-mutant gliomas don’t respond to IDH-inhibitor drugs (like vorasidenib) may have tumours with more advanced hypomethylation, making the drug’s intended effect - nudging cells toward a more mature, slower-growing state - harder to achieve.

• The researchers plan to investigate this hypothesis directly in future work, which could eventually inform patient selection for IDH-inhibitor treatment.

• Journal reference: Nomura M, Raviram R, Schiffman JS, et al., Nature Genetics (2026), DOI via nature.com/articles/s41588-026-02642-7.

Links: Weill Cornell Medicine report | Nature Genetics paper

5. Sugar-coated nanoparticles use glioblastoma’s own metabolism against it to cross the blood-brain barrier

Originator / Senior Authors: Dr. Oleh Taratula and Dr. Olena Taratula, Oregon State University College of Pharmacy
Co-Authors: Yoon Tae Goo, Vincent N. Cataldi, Vladislav Grigoriev, Neera Yadav, Tetiana Korzun, Chao Wang, Adam W.G. Alani
Clinical Sponsor: Oregon State University (preclinical, mouse model - no clinical trial yet)
Funder: National Cancer Institute (NIH grants R01CA237569, R37CA234006); Eunice Kennedy Shriver National Institute of Child Health and Human Development (R01HD101450, R01HD112007); National Research Foundation of Korea
Campaigning Organisation: None identified for this story
Reporting Source: SciTechDaily, 10 July 2026, reporting on the Journal of Controlled Release (published 15 June 2026)

Why it matters: The blood-brain barrier remains one of the fundamental obstacles in glioblastoma treatment; most drugs that work well elsewhere in the body simply can’t get into brain tissue in useful concentrations. This approach is clever because it doesn’t try to force a way through the barrier; it hijacks a transporter (GLUT1) the barrier already uses to let glucose through, using a sugar look-alike (mannose) to smuggle the treatment in. It then exploits the fact that glioblastoma cells are unusually hungry for that same transporter, so the treatment concentrates preferentially in the tumour rather than spreading evenly through healthy brain tissue. This is mouse data only, so it’s an early-stage proof of concept rather than something with any near-term relevance to patients - but still, it’s something else in the pipeline.

Details:

• The nanoparticles are coated densely with mannose, a sugar molecule similar enough to glucose that it’s recognised by GLUT1, the transporter brain blood vessels use to move glucose from blood into brain tissue.

• By chemically bonding mannose to cholesterol (a structural component of the nanoparticle), the researchers increased sugar-surface coverage sixfold, helping the particles compete against the much higher natural concentration of glucose in blood.

• Glioblastoma cells express GLUT1 at roughly three times normal brain tissue levels, so once nanoparticles cross into the brain, they preferentially accumulate in tumour tissue.

• The nanoparticles carry mRNA instructions for cells to produce PTEN, a tumour-suppressing protein commonly lost in glioblastoma; restoring it helps re-establish control over cell growth.

• In mice with glioblastoma, the treatment increased median survival by 50% compared with untreated animals, with tumour shrinkage across repeated dosing and no measurable organ toxicity.

• Journal reference: Goo YT, et al., “Single-ligand dual-targeting lipid nanoparticles for therapeutic mRNA delivery to glioblastoma across the blood-brain barrier,” Journal of Controlled Release (2026), DOI: 10.1016/j.jconrel.2026.115107.

Links: SciTechDaily report | Journal of Controlled Release paper

6. Campaigning context: Rare Cancers Bill implementation - Brain Cancer Justice at the table

Originator: Prospect Magazine / H/Advisers Cicero, event: “Driving Policy Forward: Implementing the Rare Cancers Bill”
Featured Campaigner: Georgina (Georgie) Maynard, co-founder, Brain Cancer Justice
Other Attendees: Cameron Miller (The Brain Tumour Charity); Scott Arthur MP (sponsor of the Rare Cancers Bill); Hugh Adams (Brain Tumour Research)
Campaigning Organisation: Brain Cancer Justice, alongside Brain Tumour Research and The Brain Tumour Charity
Reporting Source: Brain Tumour Research, “Implementation – the word for 2026”

Why it matters: This story dates from earlier than this week; but it’s important to keep an eye on campaigning platforms.

Brain Cancer Justice is a comparatively young campaigning voice in this space - co-founded by Georgie Maynard, who was diagnosed with a glioblastoma in 2023, alongside Matthew Wilson - but it’s earned a seat at exactly the kind of table that decides how legislation actually reaches patients. This event wasn’t about whether the Rare Cancers Bill passes (it has cross-party support and was nearing Royal Assent), but about the much harder question of implementation: who’s accountable for delivering it, and how. That a patient-founded organisation like Brain Cancer Justice was in the room alongside the Bill’s sponsoring MP and the sector’s largest charity is a meaningful marker of how much ground the organisation has covered in a short time - Maynard has also driven a Downing Street petition and secured parliamentary debate time on brain cancer funding.

Details:

• The Rare Cancers Bill, introduced by Scott Arthur MP via the Private Members’ Bill ballot, had cross-party and Government support and was expected to reach Royal Assent within weeks of this event.

• The event focused on three implementation questions: who should lead rare cancer research delivery; how to unlock data-sharing between cancer registries and the “Be Part of Research” registry; and how to reform UK orphan drug regulations.

• Brain Tumour Research described 2026 as “the year of implementation” - the point where announcements (a National Cancer Plan, research funding updates, the Rare Cancers Bill) need to translate into structural change rather than remaining goodwill.

• Separately, Brain Cancer Justice has run an active Downing Street petition campaign (“Invest in brain cancer and give rights - turn terminal into treatable”) and helped secure a parliamentary debate on brain cancer funding.

Links: Brain Tumour Research: Implementation – the word for 2026 | Rare Cancers Bill: What you need to know

That’s all for this week.

Drug discovery timelines are too slow for many people already living with brain tumours, so campaigning must also target earlier diagnosis, NHS treatment and trial access.

A note on this article, before we get to the numbers: this started as a question about drug development timelines, but it’s really about something a lot of us in this community feel and rarely say out loud. When someone is told they have a life expectancy of one to five years, and the treatment that might have changed that outcome is still ten or fifteen years from approval, campaigning can start to feel like speaking up for people who haven’t been diagnosed yet. That does not make campaigning futile; it makes the choice of campaign target vital. The tension is real, it’s documented, and it’s worth understanding - because some of these timelines can be shortened.

Why treatment development currently doesn’t keep pace with diagnosis - a summary of timelines

Across all disease areas, the average new drug takes somewhere between 10.5 and 15 years to travel from initial discovery to a patient’s prescription. Here’s roughly how that time is spent:

Only a small fraction of candidates that enter this pipline ever reach the end of it. Across oncology generally, only about 5% of drugs that reach the FDA as an investigational new drug application are ultimately approved. That’s the baseline. Brain cancer does considerably worse than that baseline.

Why brain cancer lags behind

This is the part that matters most for anyone advocating in this space: brain tumours aren’t just subject to the general problem; they’re a documented outlier, even within oncology.

• For brain cancer specifically, the historical success rate for investigational drugs is closer to 1% - roughly a fifth of the already-low oncology average.

• Between 1998 and 2014, 78 investigational brain tumour drugs reached advanced (Phase 2/3) trials. Only three were approved, and only one of those - temozolomide - extended survival.

• No systemic drug has transformed the standard treatment of classic IDH-wildtype glioblastoma since temozolomide. Vorasidenib’s 2024 approval was a major breakthrough, but for a different population: Grade 2 IDH-mutant astrocytoma or oligodendroglioma.

• The reasons are structural as well as biological: the blood-brain barrier blocks most drugs that work well elsewhere in the body, tumour heterogeneity defeats single-target approaches, and smaller patient populations mean less commercial incentive. Market size, not just biology, predicts where drug development money goes.

Two case studies that show the gap in real numbers

1. Vorasidenib: 16 years from discovery to prescription

• 2008 - The IDH1 mutation is identified by Bert Vogelstein’s team at Johns Hopkins and Duke.

• 2015 - Formal drug development of vorasidenib begins.

• 2023 - Phase 3 INDIGO trial results published in the New England Journal of Medicine.

• August 2024 - FDA approval, granted with every acceleration tool available: orphan drug status, fast track, breakthrough therapy designation, and priority review.

Sixteen years from the causal discovery to patient access - nine years even from the formal start of development - despite the drug receiving every regulatory fast-track option that exists.

2. CAR-T cell therapy: still zero approvals for brain tumours, nine years on

• 2010 - First successful CAR-T trial, in blood cancer.

• August 2017 - First FDA approval (Kymriah), for paediatric leukaemia.

• 2026 (now) - Until June 2026, there were no approved CAR-T therapies for solid tumours. China’s approval of satri-cel for gastric cancer changed that, but there is still no approved CAR-T therapy for any brain tumour.

The brain tumour CAR-T work covered in recent editions of BTNL - the McMaster GPNMB study, the Children’s National paediatric trial - remain at the preclinical or Phase 1 stage.

The honest tension

Every acceleration mechanism that exists - breakthrough therapy designation, priority review, adaptive trial designs, accelerated approval based on surrogate endpoints - was used on vorasidenib, and it still took 16 years. These tools shave months off the regulatory review at the very end of the process. They don’t touch the years of biological validation that have to happen before a drug can safely be administered to a human being. That’s the part that’s very hard to compress - and it’s also the part that a prognosis of one to five years simply doesn’t leave room for.

This is, I think, part of the frustration so many of us feel - that patient advocacy inevitably ends up speaking for future patients rather than present ones - is a real and recognised tension in our community, not just a feeling. It’s also why campaigning on drug discovery timelines can’t be the only lever we pull.

What can move faster, right now

In the immediate term, the parts of this system that can be shortened for people currently living with a brain tumour aren’t in the lab - they’re in what happens after a drug already exists:

• Diagnosis speed. The nanopore sequencing work covered from BNOS 2026 can cut a multi-week wait for molecular diagnosis down to about a single day - no new drug required, just wider adoption of existing technology.

• NHS access speed. Even an approved, funded drug like vorasidenib can currently take up to 90 days to reach a patient at a given trust, purely because of workforce capacity and funding-process bottlenecks - a systems problem, not a science problem.

• Trial access. Faster, better-signposted routes into clinical trials mean patients can access experimental treatments years before they’d otherwise reach approval - this is arguably where campaigning effort has the most immediate benefit for people diagnosed today.

None of this closes the gap entirely. But it reframes where the campaigning energy can do the most good for the people ‘in the room’ right now, rather than only for the patients who haven’t yet been diagnosed.

Moving on to the hopefully not-too-distant future: reducible numbers - the cost of shifting the timelines

Those five durations in the table above are not laws of physics. They’re not the speed of light or the existence of gravity. They’re the product of how much parallel capacity is funded at each stage - and that means, at least partly, that they can be reduced. Below is a worked model for the Discovery & Preclinical phase specifically (that’s the first duration in the table above), built from published R&D cost and timeline data and anchored against a real precedent for buying time with money. It’s presented as a model, not a peer-reviewed finding, but it’s a concrete, defensible number, not guesswork.

Breaking the phase into its four real components

“Discovery & preclinical” isn’t one activity. It’s four, and they don’t all respond to funding the same way:

That last row matters more than the other three combined for how we should campaign on this. Toxicology studies have a genuine biological floor; you cannot observe a 90-day safety signal in 30 days, no matter how well-funded the lab is. Money can only buy away the queueing-up time before those studies start, not the observation window itself. The first three rows, by contrast, are substantially about capacity: more parallel chemistry teams, more screening throughput, more compute for AI-driven candidate prediction. That’s where the campaigning case is strongest.

The formula: ‘project crashing’

This is a genuine, named technique from engineering project economics - project crashing, or time-cost trade-off analysis, from Critical Path Method (CPM) project management - adapted here to a drug discovery pipeline:

Cost Slope (S) = (Cc − Cn) / (Tn − Tc)

Cost(T) = Cn + (Cc − Cn) × [(Tn − T) / (Tn − Tc)]ᵖ

Where Tn is the Normal Time (today’s 3–6 years), Cn is the funding currently spent to achieve it, Tc is the Crash Time - the fastest this phase could go with effectively unlimited money - and Cc is what that would cost. The exponent p (greater than 1) captures a real, observed pattern in every crashing curve: cost accelerates sharply as you approach the floor (the baseline, irreducible timespan). I know this is tedious, but we don’t have to fully understand the mathematics. Suffice to say that early money buys a lot of compression cheaply; the last few months cost disproportionately more.

Anchoring it to something real: Operation Warp Speed

The closest real-world proof that this mechanism works is Operation Warp Speed. It didn’t skip a single required step - it ran normally-sequential stages in parallel (animal toxicology alongside Phase 1 human trials, manufacturing before efficacy data even existed) - and compressed an 8–12 year vaccine development timeline to under one year, for roughly $18 billion across a portfolio of eight candidates. That is the mechanism in action: money buys parallel capacity, not a suspension of biology.

Working the numbers

Using a Normal Time of 4.5 years (the midpoint of 3–6), a Crash Time of 1.5 years (justified by the toxicology floor above), and a Crash Cost of six times Normal Cost - a multiplier in line with the a large funding increase Operation Warp Speed represented over business-as-usual R&D spend - here’s what the funding curve looks like for an illustrative £1m baseline:

Just over double the funding could plausibly cut preclinical time by a third. Reaching the absolute floor costs six times as much - because the last mile fights real biology, not just bureaucracy.

For context, published estimates put the real direct cost of taking a single candidate through preclinical development at roughly £15–40 million, not £1 million - the £1m figure here is a simplified illustrative baseline chosen to make the multiplier easy to follow. The multiplier itself (roughly 2-2.5× to cut a third off the timeline, roughly 6× to reach the floor) holds regardless of which baseline figure is used, since it’s a ratio rather than an absolute.

Why this should give hope

A number you can name is a number you can campaign on. “It takes years, that’s just how it is” gives an advocacy organisation nothing to ask for. “Cutting this timeline by roughly a third would plausibly require around two to two-and-a-half times current funding for the reducible components.” gives campaigners an actual target: a funding ask, aimed at a specific bottleneck, with a real-world precedent behind it. It doesn’t erase the tension of watching someone you love face a one-to-five-year prognosis against a multi-year development timeline. But it replaces helplessness with a number - and a number is something a campaign can move.

Editorial note: this model combines published preclinical cost/timeline data with the Operation Warp Speed precedent as an empirical anchor for the funding multiplier. No peer-reviewed dataset ties directly to preclinical duration specifically for brain tumour programmes - this is presented as a transparent, working model rather than an established finding.

Sources & Further Reading

• Friends of Cancer Research — Drug Approval Process

• N-Side — Average Time to Bring a Drug to Market

• Ivy Brain Tumor Center — Glioblastoma Clinical Trials for New Treatments

• Ivy Brain Tumor Center — The Challenges of Treating Glioblastoma

• Johns Hopkins Technology Ventures — 10 Innovations in 10 Years: Voranigo

• National Cancer Institute — Fundamental Research Paved the Way for Vorasidenib

• FDA — Vorasidenib Approval Announcement, August 2024

• PMC — FDA Approval Summary: Vorasidenib (development timeline detail)

• Novartis — First FDA Approval for a CAR-T Cell Therapy (Kymriah), 2017

• MDPI Cancers — Advancing CAR T-Cell Therapy in Solid Tumors

• Wikipedia — Operation Warp Speed (overview, funding, timeline)

• U.S. GAO — Operation Warp Speed: Accelerated COVID-19 Vaccine Development Status

• Federation of American Scientists — How to Replicate the Success of Operation Warp Speed

• Knowledge Portal — R&D Costs (preclinical/phase cost breakdowns)

• arXiv — Valuing Pharmaceutical Drug Innovations (discovery-stage cost estimates)

Additional Reporting Source:

AOL / MSN (syndicated), 1 July 2026, ‘Brain cancer breakthrough as new study finds cell therapy could target glioblastoma’

Additional Contributor:

Dr Karen Noble, Director of Research and Policy, Brain Tumour Research

Why it matters:

The original coverage of this story focused on the science; this syndicated piece adds a UK patient-advocacy voice and some sobering context on just how far this preclinical work still has to travel before it reaches patients. It is worth remembering this is the same McMaster/King’s College London study covered in Story 4 of most recent edition (5th July) Brain Tumour News Links, but the added reaction is useful for readers who engage with the campaigning side of this Substack as much as the science.

Details:

• Glioblastoma patients typically survive only 12 to 18 months after diagnosis, underscoring why any credible new therapeutic avenue draws attention.

• Dr Karen Noble of Brain Tumour Research noted that around 3,200 people are diagnosed with glioblastoma in the UK each year, and just 4% survive five years or more.

• Dr Noble welcomed the CAR-T results as “really promising,” pointing to CAR-T’s track record in other cancers as grounds for hope this could be translated relatively quickly - but stressed that laboratory breakthroughs only help patients once they reach UK clinics.

• She used the findings to renew Brain Tumour Research’s call on government for a more attractive financial and regulatory environment to accelerate the path from discovery to treatment.

Links:

AOL report, 1 July 2026

Ok, next full edition - Sunday 12th July