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! 

Last year, Curtana Pharmaceuticals shared two publications highlighting CT-179's ability to target OLIG2 in Sonic Hedgehog medulloblastoma. Now, a new study published in the Journal of Clinical Investigation reports that CT-179 may help overcome immunotherapy resistance in glioblastoma (GBM). OLIG2 expression is common in GBM, especially in the proneural and mesenchymal subtypes. The study identifies OLIG2 as a driver of immune resistance by suppressing a signaling molecule called CXCL10, which is needed to attract CD8+ T cells into the tumor. By inhibiting OLIG2, CT-179 restores CXCL10 expression, increasing T cell infiltration and helping to convert "cold" tumors into more immune-responsive ones. While immunotherapy evasion in GBM is notoriously complex, preclinical mouse models have shown that a synergistic combination of CT-179 with anti-PDL-L1 therapy extended survival when compared to either treatment alone. Curtana is now preparing to launch a Phase 1 multi-site clinical trial in Australia (the OPAL study) to establish human safety and tolerability of CT-179. 

Our next webinar will be on Tuesday, March 3rd at 7pm ET with Dr. Robert Aiken on: "Clinical trial of a new individualized dendritic cell vaccine for treatment of newly diagnosed glioblastoma." To join, visit virtualtrials.org/webinar!

Mark your calendars for 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." 

In a new study published in Science Translational Medicine, researchers led by Dr. Hui Li at the University of Virginia School of Medicine report a first-in-class small-molecule inhibitor targeting AVIL, an oncogene previously shown to drive glioblastoma growth. Discovered through high-throughput screening, the molecule crosses the blood–brain barrier and suppressed tumor growth in multiple cell and mouse models. AVIL is mostly absent in normal brain tissue, and the molecule did not significantly affect healthy brain cells in preclinical studies, supporting its potential selectivity. While the current molecule form is not yet a finished drug, we're eager to see these researchers finalize chemical optimization of the compound and move into human trials within the next year or two.

Novocure's Tumor Treating Fields (TTF) technology first earned FDA approval for recurrent glioblastoma in 2011 and for newly diagnosed glioblastoma in 2015 (Optune Gio). While some in the neuro-oncology community have been skeptical of TTF, the technology went on to gain FDA approval for malignant pleural mesothelioma in 2019 and for metastatic non-small cell lung cancer in 2024 (Optune Lua).

This past week, Novocure received FDA approval for TTF for locally advanced pancreatic cancer, used in combination with gemcitabine and nab-paclitaxel (Optune Pax).

While TTF by itself is not a cure for brain cancer, the steadily expanding list of FDA-approved indications reinforces that this technology has demonstrated meaningful clinical efficacy across multiple solid tumor types. 

The GIANT trial has begun enrolling in the United States to test whether neoadjuvant immunotherapy can improve outcomes for people with newly diagnosed glioblastoma (GBM). Led by Dr. Mustafa Khasraw at Duke University, GIANT is a Phase 2 trial evaluating the immune checkpoint inhibitors nivolumab and relatlimab given before surgery, followed by these agents with standard radiotherapy and temozolomide afterward.

Unlike most prior GBM immunotherapy studies, this trial focuses on giving immunotherapy early (before and after initial surgery) based on the idea that an intact, treatment-naive tumor might be more responsive to immune stimulation. Interest in the trial has been heightened by high-profile patient advocates and researchers, including world-renowned melanoma expert Professor Richard Scolyer, who has lived well beyond typical expectations after his own GBM (MGMT unmethylated) diagnosis. More info on the trial can be found HERE.

Good news! The National Comprehensive Cancer Network (NCCN) patient guidelines are free, evidence-based resources written in plain language that reflect expert consensus cancer treatment guidelines used by clinicians worldwide to support decision-making. NCCN usually updates the patient guidelines for each cancer category every few years, but they just recently announced that all patient guidelines will be updated annually to better keep pace with advances in cancer care and expand access across multiple languages. This is a positive step forward; the last NCCN patient guidelines for gliomas was issued in 2024 (see here).

Researchers from the University of Michigan have reported a new metabolic vulnerability in pediatric ependymoma, a common childhood brain tumor that remains incurable with current therapies. Standard treatment today is maximal surgical resection followed by radiation, with chemotherapy offering limited benefit and no approved targeted therapies. In this new study, the investigators showed that most supratentorial ependymomas driven by the ZFTA-RELA fusion protein depend on production of itaconate, a metabolite usually made by immune cells. They found that tumor cells generate itaconate via the enzyme ACOD1, and that itaconate and ZFTA-RELA form a feed-forward loop that promotes tumor growth through glutamine metabolism. Blocking this pathway reduced fusion protein levels and shrank tumors in mouse models, providing the first evidence that ZFTA-RELA-driven ependymomas can be targeted indirectly. The researchers are now working with the Pediatric Neuro-Oncology Consortium (PNOC) to develop a clinical trial targeting the itaconate pathway.

A promising new pediatric brain tumor trial that will test personalized mRNA cancer vaccines is opening across multiple sites in Australia. The PaedNEO-VAX trial, funded by Providence Therapeutics alongside the Australian government and philanthropic partners, will enroll children and adolescents with recurrent or progressive high-grade glioma, diffuse midline glioma, medulloblastoma, and ependymoma.

Using tumor genome sequencing, each child’s cancer will be analyzed to identify individualized targets, followed by manufacture of a custom mRNA vaccine using Providence’s platform, with an anticipated turnaround of about 10 weeks from enrollment to dosing. Phase I will assess safety and dosing, and Phase II will evaluate progression, survival, and quality of life outcomes. The trial builds on Providence’s prior compassionate-use experience, including treatment of the company founder’s son with a personalized mRNA vaccine.

Providence has expressed interest in working with partners in the United States and Canada, including patient advocacy organizations, to explore launching a similar North American trial.

If you are a brain or spinal cord tumor survivor, living in the NY/NJ/CT area, you are eligible to apply for a Making Headway college scholarship. In 2026, Making Headway will offer at least 15 scholarships, each worth up to $5,000. A special $15,000 scholarship will also be given as part of the 7th annual Michael Schwartz Making Headway Scholarship.

To be eligible for consideration, each applicant must:
1. Be a brain or spinal cord tumor survivor (diagnosed before the age of 18)
2. Reside in the New York City metropolitan area (including New Jersey and Connecticut)
3. Have been admitted to, or be currently attending, a two- or four-year college or vocational program.

For additional requirements, to download the application, or apply online, visit http://makingheadway.org/scholarship.

The deadline for the applications to be submitted in May 1, 2026. If you have any questions, please email Jennifer@MakingHeadway.org.

Kids and adults living with brain tumors - we invite you to submit original painting/drawings through your unique lens. Selected pieces may be featured in our upcoming 13th edition of the Musella Foundations Brain Tumor Guide to help raise awareness and inspire others facing similar paths.

Please submit your high resolution image that will appear in black & white to: Musella@virtualtrials.org. 

We're excited to share that the Musella Foundation Copayment Assistance Program is now OPEN to new patients again! After being temporarily closed to new applicants, we are once again accepting applications and helping patients access needed treatments. 

What's new? 
- We've added the drug Modeyso to our list of covered treatments
- We've increased the income eligibility guidelines, allowing more families to qualify for assistance

Cancer is hard enough — worrying about treatment costs shouldn't be part of the burden. Our goal is to reduce financial barriers so patients can focus on their care. For more information and to apply, go to braintumorcopays.org.

Another encouraging update on GB13, a targeted immunotoxin that selectively kills brain tumor cells that express the IL13Ra2 receptor, which is found on many DIPG tumors but is largely absent from normal brain tissue.

In a newly published preclinical animal study, GB13 significantly reduced tumor size and improved survival across multiple DIPG models. The results were especially compelling when GB13 was given before and alongside radiation therapy. Notably, durable (and in some cases curative) responses were observed in human tumor–derived DIPG models. Researchers found that GB13 interferes with tumor cells’ ability to repair radiation-induced DNA damage, making radiation more effective at killing cancer cells. This radiosensitizing effect was consistent across multiple patient-derived DIPG cell lines and validated in both immunocompromised and immunocompetent mouse models. Together, these findings strongly support Targepeutics’ planned IND submission and upcoming Phase I clinical trial, with an expansion Phase II component, which will evaluate GB13 in children with DIPG.

As we’ve shared previously, The Musella Foundation helped support the early research behind GB13 more than a decade ago. It’s exciting to see this work continue to move steadily toward the clinic!