- Webinar tomorrow!
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. - $434,000 Awarded for Brain Tumor Research in 2026
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. - Advanced Brain Cancer Survival Rates Double with LITT
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. - Tumor-brain crosstalk restrains cancer immunity via a sensory-sympathetic axis
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. - Tumor Treating Fields (TTFields) Therapy to be Covered for Patients with Newly Diagnosed Glioblastoma in British Columbia
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. - FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases
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." - Musella Foundation Copay Program Closing Soon
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!
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