We are proud to announce that we have awarded a new $50,000 venture philanthropy grant to AVIL Therapeutics to support promising research aimed at developing a targeted therapy for glioblastoma (GBM). Our venture philanthropy model helps accelerate research projects that show both scientific rigor and clear potential for patient benefit. By supporting early-stage but transformative ideas, we help bridge the gap between discovery and future clinical trials. 

This grant supports preclinical work focused on AVIL (advillin), a newly identified cancer driver that appears to play a critical role in glioblastoma growth and survival, while being largely absent from normal brain tissue. Specifically, the funded research will focus on advancing preclinical validation and lead optimization, with the goal of generating the data needed to support future clinical development. By emphasizing rigorous science and translational relevance, this work aligns closely with our mission to accelerate progress toward more effective treatments for patients and families affected by GBM. 

We are also very proud to announce the award of a $50,000 venture philanthropy grant to KiraGen Bio to support groundbreaking work in engineered CAR-T cell therapy for glioblastoma (GBM). KiraGen is developing off-the-shelf CAR-T cell therapies that are engineered to 'tune out' immune suppressive signals in the tumor microenvironment, thus helping the CAR-T treatment remain active and effective longer without increasing toxicity. 

This grant will support a study to determine which of KiraGen’s lead 6-knockout allogeneic CAR-T variants delivers the most durable anti-tumor function when faced with realistic GBM tumor microenvironment suppression. Using patient-derived GBM tumoroids that preserve native tumor architecture and immune suppressive signaling, the research team will test these engineered cells against conventional CAR-T to decide which configuration is best positioned to advance toward a clinical trial. By supporting this work, we hope to move the next generation of engineered cell therapies closer to patients and families affected by GBM.

The FDA has granted breakthrough therapy designation to plixorafenib, an oral targeted drug, for patients with recurrent high-grade gliomas with a BRAF V600E mutation. This designation allows closer collaboration with the FDA and is intended to shorten development time.

Early clinical results show encouraging activity, particularly in brain tumors. In a small predefined subgroup of 9 patients with BRAF V600E-mutated primary CNS tumors (including high-grade gliomas) who had not received prior similar targeted therapies, 67% had tumor shrinkage and more than 75% experienced clinical benefit (shrinkage or stable disease).

Across all BRAF V600-mutated solid tumors, the response rate was 42%, with a median response duration of 17.8 months and over 70% experiencing clinical benefit.

The drug has shown a generally favorable safety profile, and an ongoing phase 2 trial is further evaluating its effectiveness in BRAF-mutated cancers, including brain tumors. While these results are promising, larger studies are needed to confirm efficacy and response durability in high-grade glioma.

Researchers have been working to overcome a major limitation in glioblastoma research: traditional lab models don’t accurately reflect how tumor cells infiltrate the human brain. At institutions such as Weill Cornell and others worldwide, scientists are now using patient-derived brain organoids (three-dimensional lab ‘mini-brains’ grown from a patient’s own tumor cells) to study tumor behavior and test hundreds of existing drugs or combinations in a personalized way. These organoid platforms can better mimic each patient’s tumor genetics, microenvironment, and patterns of invasion, and are showing promise for identifying therapies that might be more effective than standard treatments alone. While this technology has not yet been used in the US to guide therapy in large clinical trials, several research centers are actively developing organoid screening systems and working with regulatory agencies to design early-phase studies. We hope that, in coming years, organoid-based drug testing could help tailor treatments to individual patients and accelerate development of new therapies for high-grade brain cancers.

The National Institute for Health and Care Excellence (NICE) has published final guidance approving vorasidenib (brand name Voranigo) for prescription on the NHS in England and Wales for people aged 12 and older with IDH-mutant low-grade gliomas, including astrocytoma and oligodendroglioma. The treatment will be made available through the Cancer Drugs Fund, offering faster access while routine NHS funding is established.

Vorasidenib, which received MHRA marketing authorization in October 2025, was shown in the INDIGO trial to slow tumor growth and delay the need for chemotherapy or radiotherapy, helping patients maintain quality of life for longer. NICE’s positive recommendation reflects both the clinical evidence and the strong support from patients, families, and charities advocating for access to this much-needed therapy. This is only the second new NHS-approved drug for brain tumors since temozolomide in 2006, marking a meaningful step forward for patients and families affected by low-grade gliomas.

This is a nice review article summarizing recent advances in immunotherapies for glioblastoma, including leading strategies to overcome treatment resistance and the immunosuppressive tumor microenvironment. One of the authors, Dr. Michael Lim, is a 2026 Musella Foundation grant recipient!

Great to see Forbes covering the Phase 2 sonodynamic therapy trial for newly diagnosed glioblastoma; our recorded webinar with more details is available HERE. 

The current World Health Organization (WHO) brain tumor classifications already incorporate one molecular feature (CDKN2A/B homozygous deletion) that can upgrade an IDH-mutant astrocytoma to grade 4, even in the absence of classic high-grade histologic features. However, this new study shows there are several additional molecular alterations that independently predict worse survival in IDH-mutant astrocytoma and therefore may warrant inclusion in grading criteria.

Across two large cohorts of roughly 1,200 patients, the authors found that CDK4 amplification, CCND2 amplification, PDGFRA alterations, PIK3R1 mutations, MYCN amplification, and EGFR alterations are all associated with significantly poorer outcomes in IDH- mutant astrocytoma. This is important, because roughly 18% of tumors currently classified as grade 2 or 3 harbor at least one of these high-risk molecular features.

Clinically, these tumors fall into an intermediate-risk category, with survival outcomes worse than standard grade 2 or 3 tumors but still better than grade 4 disease. Median overall survival in this group was approximately 67 to 82 months, compared to 135 to 141 months for typical grade 2 or 3 tumors and 35 to 45 months for grade 4 tumors. 

For patients, the key implication is that a tumor labeled as grade 2 or 3 may still behave more aggressively if these molecular features are present. For clinicians, the findings support consideration of expanding molecular grading criteria to include these additional alterations, as they provide independent prognostic value, help identify patients who may benefit from closer monitoring or more aggressive upfront treatment, and improve risk stratification for clinical trials.

Hoops for Hannah is a March Madness bracket contest that raises funds for brain tumor research in memory of Hannah Taylor.  Participants have donated over $90,000 to brain tumor research through Hoops For Hannah since Hannah's passing in 2012. This cause is worth fighting for!

There is no entry fee, as Hannah's family generously provides a $1,000 First Prize; donations to Brain Tumor Research are not required but gratefully accepted. 100% of all donations go to the Musella Foundation! For details to enter, click here.

The Musella Foundation is working on an updated edition of our Brain Tumor Guide for the Newly Diagnosed. The final chapter, titled "Hope for the Future," will live on our website so that it can be updated regularly. To read it, click here!   

A new study shows adding berzosertib (an ATR inhibitor) to radiation appears to make H3K27-altered diffuse midline glioma more sensitive to treatment by blocking tumor DNA repair, leading to greater tumor cell death in preclinical models. This suggests a promising strategy to enhance radiotherapy effectiveness, but it remains experimental and requires clinical trials to confirm safety and benefit for patients.

In this Northwestern study, researchers found that glioblastoma (GBM) tumors 'hijack' microglia metabolism by exploiting a fructose transporter called GLUT5, causing the microglia (the brain's resident immune cells) to take up and metabolize fructose in a way that suppresses anti-tumor immunity. To test its role, they used genetically engineered mice lacking GLUT5.

In multiple mouse GBM models, GLUT5-deficient mice showed significantly reduced tumor growth and improved survival, indicating this pathway is required for tumor progression. The microglia became more inflammatory and less immunosuppressive, and there was greater activation of a broader immune response, including increased antigen presentation, higher inflammatory signaling, and shifts toward more active T cell responses (including CD8+ T cells).

Our Brain Tumor Copayment Assistance Program is now closed to new patients. 

Thanks to the generosity of our donors, 71 patients were approved this year for grants of up to $5,000 each - totaling $355,000 in assistance to help patients afford lifesaving treatments.

We will continue paying claims for all patients who currently have active grants.

Because this program depends entirely on donations, we never know exactly when we will be able to reopen to new patients, but we will announce it as soon as more funding becomes available. 

If you would like to help patients access lifesaving treatments, please consider making a donation: https://virtualtrials.org/donate.cfm

Please join us tomorrow, Tuesday, March 3rd at 7pm ET for a webinar with Dr. Robert Aiken on: "Clinical trial of a new individualized dendritic cell vaccine for treatment of newly diagnosed glioblastoma." To join, visit HERE. 

Thanks to the generosity of our donors, the Musella Foundation has awarded $434,000 in research grants so far in 2026, supporting innovative projects in glioblastoma, DIPG/DMG, immunotherapy, imaging, and patient-centered research.

From cutting-edge laboratory science to real-world patient support, these grants are helping move promising ideas closer to meaningful treatments. See the full list of grants and projects: 

In addition to research funding, we have also given out $280,000 in grants to patients so far this year, through our Musella Foundation Copayment Assistance Program. In order to continue this incredible momentum in accelerating the search for a cure for brain tumors while providing immediate, life-changing support to patients and their families, we need your help! To donate, please click HERE. If you are interested in helping us fundraise, please email musella@virtualtrials.org.

This is an important study from Dr. David Tran, et al. In this Phase I/IIb clinical trial, laser interstitial thermal therapy (LITT) was combined with the immune checkpoint inhibitor pembrolizumab for patients with recurrent high-grade gliomas (HGG), including glioblastoma. All patients were at least at their second recurrence, and several had reached their third (or greater) recurrence at time of entering the trial. LITT ablates tumor tissue with heat and temporarily opens the blood-brain barrier, but this study found that it also activates non-classical monocytes, priming the immune system so pembrolizumab can stimulate CD8+ T cell proliferation, clonal expansion, and memory responses. This approach, when compared with conventional non-LITT surgery followed by pembrolizumab, more than doubled median overall survival (OS) from 5.2 to 11.8 months and improved median progression-free survival (PFS) from 1.6 to 4.5 months in the per-protocol population (n = 39). Similarly, in the GBM (IDH-wildtype) sub-population (n = 34), median OS was doubled from 4.8 to 11.1 months, and median PFS increased from 1.7 to 3.9 months. Among all HGG patients in the per-protocol population treated with LITT + pembrolizumab, 42% were alive at 18 months, and over one-third survived more than 3 years.

Interesting new research has identified a precise neural circuit that tumors exploit to suppress the immune system. Using mouse models of lung adenocarcinoma, the researchers showed that tumors activate the vagus nerve, a major sensory pathway from organs to the brain. Signals travel along vagal sensory fibers to the brainstem, which then sends stress-related sympathetic signals back to the tumor microenvironment. These signals cause immune cells called macrophages to release chemicals that block T cells from attacking the cancer. Disrupting this vagal-to-sympathetic loop (genetically, pharmacologically, or through neural modulation) slowed tumor growth and restored anti-tumor immunity. While these preclinical experiments were in lung cancer, the findings may be relevant to brain cancers, where tumors are embedded within dense neural networks and may similarly hijack local or systemic nerve circuits to evade immune attack. Researchers such as Dr. Michelle Monje have previously shown in brain tumor models that neural activity can directly influence tumor growth and the immune microenvironment, so there is increasing interest in the idea that modulating neural signals could be a worthwhile strategy to improve immunotherapy for brain tumor patients.

British Columbia has become the first Canadian province to cover Tumor Treating Fields (TTFields) for adults with newly diagnosed glioblastoma (GBM), more than three years after Health Canada approved the treatment in 2022. TTFields, which has been shown to extend median survival for newly diagnosed GBM, is already reimbursed in multiple countries worldwide, and we hope to see the rest of Canada’s provinces follow BC’s lead so more patients have affordable access to this therapy.

The FDA just issued draft guidance outlining a new approval pathway for highly individualized therapies designed to treat ultra-rare diseases. The proposal introduces what the agency calls a "Plausible Mechanism Framework," aimed at situations where traditional large randomized trials are not feasible because patient populations are very small.

The draft guidance focuses on genome editing and RNA-based treatments, such as antisense oligonucleotides, that directly target a specific genetic or molecular abnormality causing disease. Instead of requiring large trials, the FDA would allow approvals based on strong biological rationale, well-documented natural history data, confirmation that the therapy successfully hits its intended target, and evidence of clinical benefit or validated biomarkers. The agency also suggests that related mutation-specific versions of a therapy could potentially be evaluated under shared master study protocols.

For the brain cancer community, this could be an important development. As tumors like glioblastoma are increasingly divided into smaller molecular subtypes (e.g. H3 G34 diffuse hemispheric glioma), some patients have rare or even unique mutations that are difficult to study in large trials. A more flexible, common sense regulatory approach could help accelerate development of precision therapies for these small groups while maintaining safety and scientific rigor.

The draft guidance, titled "Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause," is open for public comment for 60 days following publication in the Federal Register. 

To submit a public comment, visit: https://www.regulations.gov, search for the guidance title and click "Comment."

The Musella Foundation Copayment Assistance Program has given out more than 55 grants so far in 2026. Unfortunately, the program is now running low on funding and will close to new and renewal applicants soon. If you have been thinking of applying, go to braintumorcopays.org and follow the directions to send in an application!