The National Comprehensive Cancer Network (NCCN) has published a brand new, free guide to help people caring for loved ones with cancer. The guide offers practical advice on providing emotional support, managing medical care, handling finances and household responsibilities, navigating legal concerns, and maintaining one's own well-being as a caregiver. It is for caregivers across all cancer types, but it specifically mentions the Musella Foundation as a resource for brain tumor patients and their families!

Safusidenib is an investigational oral drug that crosses the blood-brain barrier and targets IDH1 mutations. The randomized, placebo-controlled Phase 3 SIGMA trial (NCT05303519) is now at the stage where they are evaluating the drug as maintenance therapy for patients with high-risk, IDH1-mutant astrocytoma following radiation and temozolomide. An exploratory cohort is also studying patients with grade 3 IDH1-mutant oligodendroglioma who have not yet received radiation or chemotherapy. The trial began enrolling in 2023, initially at sites in the United States, Australia, China, Canada and Japan. Now, new sites have been added across Europe.

Earlier this year, updated results from a separate Phase 2 study of safusidenib for previously untreated grade 2 IDH1-mutant glioma were encouraging, with a 52% confirmed response rate and 79% of patients remaining progression-free at three years. However, the Phase 3 SIGMA trial is still active and has not yet reported results. 

Researchers at UCSF have developed a CAR T-cell therapy designed to activate its tumor-targeting machinery only after reaching the brain. Unlike conventional CAR T cells, which are already equipped to recognize and attack tumor cells when infused, this approach uses a two-step system. The cells first detect brevican, a protein found in the brain, which switches on their ability to recognize and attack tumor cells. In mouse models, the treatment eliminated diffuse midline glioma (DMG) tumors, and remained active enough to attack tumors introduced weeks later. The results are still preclinical, by they hope to begin first-in-human clinical trials in early 2027.

 This is a fun fundraiser.  We receive $1 for each person who joins, so ask all of your people to join!   

 This is a real head scratcher - why did those with a worse prognosis do better than those with the better prognosis?
The drug clearly has an effect.  We need further research to see if a combination approach targeting the other mutations they mention may make a big response.

 We added vincristine and procarbazine to the list of drugs we cover in the Copay Assistance program!

Our brain tumor copayment assistance program is now open to new and renewal patients. This program can help cover copay costs for: Optune Gio, Avastin, Temodar, Lomustine, and Modeyso, as well as their generics. If you think you may need help, apply ASAP as the program often closes quickly due to high demand. Go to braintumorcopays.org for details and to apply!  

In a recent prospective pilot study, published in the Journal of Neuro-Oncology, researchers compared MRI-based and FET-PET/MRI-based radiation target volumes in 30 patients with newly diagnosed high-grade gliomas. Contrast-enhanced MRI highlights areas where the blood-brain barrier is disrupted, while FET-PET uses a radioactive amino acid tracer to identify areas of increased amino acid transport associated with glioma, potentially revealing tumor beyond the areas of contrast enhancement. FET-PET/MRI identified significantly larger target volumes, with discrepancies between the radiation approaches in more than half of patients. The findings suggest that FET-PET may help refine radiation target planning, but whether this improves outcomes remains uncertain. In another randomized trial in Europe (the GLIAA trial), published earlier this year in The Lancet Oncology, FET-PET-guided re-irradiation did not significantly improve progression-free or overall survival compared with contrast-enhanced MRI-guided re-irradiation in patients with recurrent glioblastoma. These findings highlight the urgent need for more research to determine which patients and treatment settings may benefit from FET-PET-guided planning.

This Duke phase 1 trial evaluated D2C7, an experimental therapy that targets EGFR and EGFRvIII proteins on glioma cells and delivers a toxin intended to kill them. The treatment was delivered directly into the tumor through convection-enhanced delivery (CED) and resulted in a median overall survival of 7.9 months among 72 patients with recurrent glioblastoma. Treatment-related side effects were mild to moderate, and exploratory analyses identified biomarkers associated with longer survival in some patients.

While this initial study was meant only to establish safety and dosing, Duke has already expanded on this work and is currently testing D2C7 together with 2141-V11, an experimental immune-stimulating anti-CD40 antibody. In a November 2025 conference abstract reporting preliminary results from 27 recurrent glioblastoma patients, median survival was 16.4 months with the combination approach. Treatment included a single convection-enhanced delivery treatment course with both agents, followed by repeated subcutaneous injections of 2141-V11 near the lymph nodes in the neck. No treatment-related grade 4 or 5 adverse events were reported. These early findings are encouraging, and we are eager to learn further results from the combined approach.

Initial results from the SURVIVE randomized, placebo-controlled trial of SurVaxM added to standard of care treatment for newly diagnosed glioblastoma (GBM) showed that, among 233 evaluable patients, median overall survival was 22.8  months with SurVaxM versus 20.3 months with placebo. This survival difference did not reach statistical significance. However, in the prespecified subgroup of patients aged 65 and under, the median survival difference of 24.2 months versus 20.2 months was statistically significant (p = 0.019). Further, the 30-month overall survival rate was 42% with SurVaxM versus 18% with placebo in this younger subgroup. The trial enrolled adults with newly diagnosed GBM, good functional status (Karnofsky score of at least 70), minimal residual tumor after surgery (no more than 1 cm³ of enhancement on MRI within 72 hours), and no progression after standard radiation and temozolomide. 

SurVaxM was generally well tolerated. Common side effects included injection-site reactions (48%), pain (41%), redness (39%), itching (38%), swelling (29%) and flu-like symptoms (13%). While the trial did not meet its primary endpoint, the results are particularly encouraging for patients 65 and under. It is worth noting that the placebo group lived longer than the 16-month historical benchmark, making the study's comparison more challenging. SurVaxM remains investigational, but MimiVax plans to discuss the results and potential expedited approval pathways with the FDA.

'Butterfly glioblastoma' is when a GBM tumor crosses the brain's midline through the corpus callosum. These tumors account for an estimated 2-6% of GBM cases and are particularly challenging to treat. Because treatment options are limited and clinical trials specifically for these patients are limited, this new trial evaluating the investigational combination of 5-ALA with low-intensity diffuse ultrasound offers an important opportunity for a group with significant unmet need. 

A window-of-opportunity trial in recurrent GBM found that neoadjuvant (pre-surgery) dual immunotherapy with nivolumab (Opdivo) and ipilimumab (Yervoy) significantly increased the number of immune cells infiltrating the tumor compared with placebo. Median overall survival was 402 days among patients who received the dual immunotherapy (including patients who received the combo both before and after surgery and those who received it only after surgery), compared with 273 days for those who got nivolumab alone. Exploratory analyses also found increased activity in blood markers associated with immune system activation for patients receiving the dual treatment. Higher levels of tumor-infiltrating immune cells and early signs of immune activation were associated with improved survival, while tumor mutational burden was not. These results show that dual checkpoint blockade can activate immune responses in GBM, and we hope to see further studies to validate whether this strategy can help improve survival outcomes. 

A phase 3 randomized trial of 157 patients found that radiation after complete surgical removal of a grade 2 atypical meningioma significantly reduced recurrence compared with observation. At five years, 80% of patients who received radiation remained disease free, compared with 64% of those who were just observed. The trial supports radiation after complete resection, but treatment decisions should still depend on individualized clinical circumstances and the balance of benefits and risks.  

In a preclinical study, researchers at The Wistar Institute in Pennsylvania found a combination that improved immunotherapy results in glioblastoma mouse models. They used low-dose axitinib to reduce oxygen deprivation (hypoxia) in tumors, which can cause immune cells called myeloid cells to suppress cancer-fighting T cells. They then added a treatment that activates a certain receptor on T cells to strengthen their ability to attack the tumor. The combination extended median survival in mice from 17 days in untreated controls to 42 days, and 40% of the mice survived long term. The surviving mice also showed evidence of lasting immune memory against the tumor. The findings suggest a potential new approach to overcoming immune suppression in GBM, but clinical trials are needed to determine whether the combination is safe and effective in humans.

Struck by Lightning: A GBM is a new podcast that follows H. Gulick, a longtime paramedic and senior FBI agent diagnosed with GBM, as he navigates treatment and life with brain cancer. Through candid conversations with his friend Tim Mayer, H shares his experiences with treatment, family, uncertainty and the realities of living with GBM, offering support and connection to other patients and caregivers.  

The Musella Foundation is working to develop a patient decision aid to help brain cancer patients and families understand the benefits and limitations of tumor treating fields (TTFields), so they can make a more informed decision about whether this treatment is right for them.

You can help! Read our short education module and then take a brief test to see how well the information was communicated. It takes about 15–20 minutes, and your answers will help us improve and standardize how information about TTFields is shared with patients and families.

We have been following Imvax and its personalized glioblastoma (GBM) vaccine, IGV-001, for several years. The early Phase 1b data caught our attention because it included a notable group of long-term survivors, with 5 of 33 patients living at least 60 months. Subsequent studies continued to show encouraging survival signals.

Now, Imvax has announced plans to move IGV-001 into a randomized Phase 3 trial in newly diagnosed GBM patients with an unmethylated MGMT promoter. The study is expected to enroll about 375 patients in the US. In the Phase 2b trial, median overall survival was 20.3 months with IGV-001 compared with 14.0 months with placebo. In the subgroup of 48 patients with unmethylated MGMT promoter, median overall survival was 14.8 months compared with 10.6 months (4.2 month difference, p = 0.0808). Imvax also reported delayed deterioration of Karnofsky Performance Status, favorable quality-of-life measures, fewer severe toxicities and a lower burden of serious adverse events for IGV-001 patients compared with placebo.

When T cells, a type of immune cell that can attack cancer, are exposed to a tumor for too long, they can become “exhausted” and lose their effectiveness. Researchers at St. Jude Children’s Research Hospital found that ZMYND8, a gene-regulating protein, helps drive this exhaustion by turning down IL-2 signaling, which helps T cells stay active. In mouse models, removing ZMYND8 helped T cells remain functional and improved their ability to control tumors, and combining ZMYND8 removal with either IL-2 or checkpoint blockade produced an even stronger response. The findings suggest that blocking ZMYND8 could potentially help overcome T-cell exhaustion and make existing immunotherapies more effective, although the research is still preclinical.

Medulloblastoma has four major molecular subgroups. In high-risk groups 3 and 4, the tumors can have changes in the way DNA is packaged and controlled. Recent preclinical research from St Jude and collaborators has identified a potential vulnerability involving a protein called KDM2B, which appears to help these tumor cells maintain their cancerous state. When researchers removed or blocked KDM2B, the group 3 and 4 tumors grew much less effectively in lab and animal models. Thus, KDM2B may be a promising therapeutic target; however, more research is needed to determine whether this mechanism can be safely and successfully targeted in patients.

For our friends in or near Massachusetts, the Harvard Medical School Center for Bioethics will host “Dignity and Defiance: The Voice of Glioblastoma” on October 1, exploring visibility, dignity, and a new ethic of care for people living with glioblastoma and the families who care for them. The free, in-person event is open to the public; register here.