Latest News

• Musella Foundation Supports New Glioblastoma Research Through Venture Philanthropy Grant to AVIL Therapeutics        

  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. 

  
  
• Musella Foundation Supports New Glioblastoma Research Through Venture Philanthropy Grant to KiraGen Bio        

  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.

  
  
• Expanding the molecular grading criteria in IDH-mutant astrocytoma        

  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.

  
  
• Brain Organoid Models Pave the Way for Precision Medicine for Glioblastoma        

  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.

  
  
• Vorasidenib approved for NHS use in England        

  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.

  
  
• Plixorafenib Receives FDA BTD for BRAF V600E-Mutated High Grade Glioma        

  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.

  
  
• Next-generation immunotherapy biologics for glioblastoma        

  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!

  
  
• Clinical Trial For Brain Cancer Treatment Has Promising Results        

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