Latest News

• Musella Foundation's Brain Tumor Guide        

  We are proud to announce that a new, updated edition of our Brain Tumor Guide for the Newly Diagnosed is now available! You can view it online HERE or order a free printed copy HERE. 

  
  
• Musella Foundation Copayment Program now closed        

  Our copay program has recently closed to new and renewal applications. We will reopen again when we are able to raise more funds. Of course, we will continue to pay claims for those who have an active grant. This program is funded entirely by donations earmarked for this purpose; if you'd like to make a donation towards this program, go to virtualtrials.org/donate and select 'Co-payment Assistance Fund' when you make a donation!  

  
  
• UAB trial shows first-in-human immunotherapy that more than doubles progression-free survival in glioblastoma patients        

  Results from the Phase 1 INB-200 study of gamma-delta T cells for newly diagnosed glioblastoma have now been published in the Journal of Clinical Oncology. While the publication provides additional details on the novel cell therapy, the most up-to-date clinical results can be found in an abstract presented at the ASCO 2026 conference.

  
  
• BPGbio Reports Encouraging Preliminary Data from Ongoing Phase 2 Study of BPM31510 in Newly Diagnosed Glioblastoma        

  Preliminary Phase 2 results suggest that BPM31510, an investigational intravenous mitochondrial therapy, may improve outcomes in newly diagnosed glioblastoma (GBM) when added to standard radiation and temozolomide. BPM31510 is designed to deliver very high levels of oxidized CoQ10 into tissues, including the brain, where it may disrupt tumor metabolism, increase oxidative stress, and trigger cancer cell death. The treatment is given together with vitamin K1 to reduce coagulation-related side effects. 

  In this single-arm Phase 2 trial, BPM31510 was given with standard chemoradiation and temozolomide. Among 39 evaluable patients, median overall survival was 19.3 months. The most encouraging results were seen in the 24 patients with MGMT-unmethylated tumors, where median overall survival reached 29.3 months. It is not yet known whether or why MGMT-unmethylated patients appeared to benefit more, and these results need confirmation in larger randomized studies. Final results from all enrolled patients are expected later this year.

  
  
• New autologous T cell therapy offers hope for children with brain tumors        

  A Phase 1 trial from Children's National in Washington, DC tested an experimental autologous (patient-derived), non-genetically engineered T-cell therapy targeting WT1, PRAME, and survivin in children with aggressive brain tumors. The study enrolled 11 children with newly diagnosed DIPG and 22 with recurrent non-brainstem brain tumors who received the treatment. The therapy was generally well tolerated, but there was one potentially treatment-related death from tumor swelling in a child with DIPG, which led to a study protocol change. Median overall survival in the DIPG group was 13.7 months. Among the patients with recurrent non-brainstem tumors, three patients (14%) remain disease-free for 31.8-51.6 months without additional treatment, including one patient who had a complete MRI response. The three long-term survivors had recurrent medulloblastoma, anaplastic ependymoma, and high-grade glioma. While this study was designed mainly to assess safety rather than efficacy, the results support further investigation of this personalized T-cell approach. 

  
  
• Anti-LAG-3 with or without anti-PD-1 in recurrent glioblastoma: a phase 1 trial        

  A Phase 1 trial enrolled 46 patients with first recurrence glioblastoma (GBM) who had previously received standard surgery, radiation, and temozolomide. The study was designed prior to 2016, so 6 of the 46 patients included had IDH mutations. The researchers tested relatlimab, an antibody that blocks LAG-3, an immune checkpoint that acts as another "brake" on exhausted T cells. Half the patients received relatlimab alone, while the other half received it with nivolumab (Opdivo), a PD-1 checkpoint inhibitor. Although Opdivo has previously failed as a monotherapy to improve survival in GBM trials, combining it with LAG-3 blockade may help overcome some of the immune suppression seen in GBM. The study's primary goal was safety, which was achieved. While the trial was not designed to prove efficacy, the one-year survival was 52.2% with the combination versus 34.8% with relatlimab alone, and researchers found evidence of increased immune-cell infiltration into tumors. It is also worth noting that the five patients who survived longer than two years were in the combination therapy group. A randomized Phase 2 trial is now underway to determine whether the combination improves outcomes.

  
  
• Postoperative gabapentinoid use is associated with improved survival in glioblastoma: A nationwide population-based study        

  A new study published in Neuro-Oncology Advances adds to growing evidence that gabapentinoids (ie, medications such as gabapentin and pregabalin commonly used for nerve pain and seizures) may have anti-tumor effects in glioblastoma (GBM). Researchers analyzed data from 11,924 adults with newly diagnosed GBM from a nationwide Turkish registry. After adjusting for clinical factors, postoperative gabapentinoid use was associated with improved overall survival (hazard ratio 0.75). The survival association was strongest in patients age 55 and older (HR 0.70), while no significant benefit was observed in patients younger than 55. Both gabapentin and pregabalin were associated with improved survival in the older subgroup.

  The findings follow a 2025 study from researchers at UCSF, Mass General Brigham, and collaborating institutions, published in Nature Communications, which reported that gabapentin use was associated with longer survival in GBM patients. Both studies are retrospective and cannot prove that gabapentin or pregabalin directly improve outcomes, but they support further investigation of gabapentinoids as potential repurposed therapies targeting the interaction between neurons and glioblastoma cells.

  
  
• Signaling Pathway May Help Predict Immunotherapy Response in Glioblastoma        

  Researchers at Northwestern have identified a biological pathway that may help explain why some glioblastoma (GBM) patients respond to immunotherapy while many do not. Using CRISPR-based screening in mouse models, they found that activation of the MAPK/RAF-MEK-ERK signaling pathway made tumors more recognizable to the immune system and improved responses to immune checkpoint inhibitors such as anti-PD-1 and anti-CTLA-4 therapies.

  The findings build on earlier observations that rare BRAF and PTPN11 mutations, which activate MAPK signaling, were more common among the small number of GBM patients who had exceptional responses to immunotherapy. The researchers found that MAPK/ERK activation increases inflammatory signals, promotes T-cell infiltration into tumors, and may help immune cells recognize and attack cancer cells.

  The team cautions that MAPK activation is necessary but not sufficient for immunotherapy response; in other words, some patients with this pathway activation still do not respond. If validated in human studies, MAPK/ERK activity could become a biomarker to help identify which GBM patients are most likely to benefit from immunotherapies.

  
  
• Compound BA-101 Reverses Glioblastoma Chemo Resistance        

  Researchers from the Hebrew University of Jerusalem and Harvard Medical School have identified a potential new strategy for overcoming temozolomide (TMZ) resistance in glioblastoma (GBM). In a study published in Cancer Medicine, investigators showed that an experimental compound called BA-101, a selective inhibitor of neuronal nitric oxide synthase (nNOS), reduced growth and invasion of resistant GBM cells and improved the effectiveness of TMZ in laboratory and mouse models.

  The researchers found that resistant GBM cells rely on increased production of nitric oxide through the nNOS pathway to survive chemotherapy. By blocking this pathway, BA-101 appeared to weaken the tumor’s defenses and restore sensitivity to TMZ. However, the approach remains experimental and will require additional preclinical testing and clinical trials before it can be evaluated in patients.