Registration is now open for the 5th Annual BrainStorm Summit, taking place September 16-18, 2026 in McLean, Virginia. The Summit brings together pediatric brain tumor families, researchers, clinicians, advocates, nonprofit organizations, and industry partners for three days of collaboration, education, research updates, and networking. With dedicated programming for children, teens, young adults, and adults, the event is designed to foster meaningful connections and advance progress against pediatric brain tumors. Learn more, register, and reserve discounted hotel accommodations at brainstormsummit.org. 

This recent review article nicely summarizes the current state of glioblastoma treatment and emerging therapeutic strategies. While it does not present new clinical data, it comprehendively covers standard therapy, immunotherapy, precision medicine, novel drug delivery approaches, and new approaches to clinical trials.

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. 

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!  

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.

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. 

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.

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.

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.

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.

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.

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.

Our Copayment Assistance program is running low on funding and will close to new and renewal applications soon. If you have been thinking of applying, go to braintumorcopays.org and follow the directions to send in an application!

The Phase 3 trial results for DCVax-L showing extended survival for both newly diagnosed and recurrent glioblastoma (GBM) were published previously in JAMA, but new additional statistical analyses of the results using propensity score matching were presented by Dr. Marnix Bosch at the British Neuro-Oncology Society (BNOS) Annual Meeting last week. The more rigorous analyses (available here) show the trial results are even better than previously reported, and that the vaccine more than doubled the 5 year survival rate for newly diagnosed GBM.

One of the biggest challenges with CAR-T therapy for high-grade gliomas has been the tumor's ability to suppress the immune system. A new study published in Nature proposes a solution by designing CAR-T cells that attack not only the tumor itself, but also the immunosuppressive cells protecting it. Rather than targeting one of the more familiar glioblastoma (GBM) markers, such as EGFRvIII or IL13Rα2, the researchers identified GPNMB as an ideal dual-purpose target because it is found on both GBM cells and tumor-associated macrophages (the immune cells that help shield the tumor from attack). By simultaneously eliminating both the cancer cells and their protective microenvironment, the treatment produced dramatic results in mouse models, including what appeared to be long-term cures in many animals. While these findings are still preclinical, the study introduces a promising new strategy for CAR-T therapy.

ATRX mutations have been recognized as a hallmark of many gliomas for years, and because ATRX is a chromatin remodeling protein, researchers have long suspected that losing its function alters how DNA is organized and which genes are switched on or off. This study from researchers at MD Anderson Cancer Center strengthens that theory by showing how ATRX loss rewires the 3D structure of chromatin, activating developmental gene programs that promote tumor growth.

The researchers found that ATRX-mutant tumors become highly dependent on the HOXA developmental gene network. They then tested a compound that blocks the interaction between HOXA and its partner protein PBX. In lab models and mice with patient-derived ATRX-mutant glioma cells, the treatment slowed tumor growth, suggesting this pathway could become a future therapeutic target. While these findings are still preclinical, they help explain why ATRX mutations promote glioma progression and identify a specific downstream vulnerability that may be easier to target than ATRX itself. 

In this small trial out of Beijing, eleven patients with newly diagnosed glioblastoma received a personalized dendritic cell vaccine, created using neoantigens unique to each patient's tumor, after surgery and standard chemoradiation. The treatment was well tolerated, as has been seen repeatedly in dendritic cell vaccine trials. Although the primary goal of the study was to evaluate safety, the median progression-free survival was 16.2 months, the 12-month overall survival was 100%, and the median overall survival had not yet been reached at the time of publication. The vaccine generated strong neoantigen-specific immune responses, and patients with the strongest immune responses tended to remain progression-free longer. While the study is too small to determine whether the vaccine improves survival, the results provide encouraging support for advancing this personalized vaccine approach into larger Phase 2 trials.

This program, brought to you by our friends at the Head for the Cure Foundation, offers financial support to individuals diagnosed with primary malignant brain tumors. To ease the financial burden, approved funds may be used to pay for clinical trial or treatment-related airfare/lodging, or specific bills, such as rent or mortgage, utilities, medical expenses, and more. Learn more about the criteria, guidelines, and how to apply here.

Coherence Neuro, a company with ties to Neuralink, has reported early human feasibility testing of its implantable brain-computer interface during brain tumor surgeries in Australia. In three patients undergoing tumor resection, the device was temporarily placed in the brain for approximately 30 minutes to assess short-term safety and its ability to record electrical activity from tumor-related brain tissue before being removed. The procedures were designed as a proof-of-concept for intraoperative use rather than a therapeutic intervention.

The company has stated that it is developing the technology for longer-term implantation in patients with brain tumors, with an initial focus on glioblastoma (GBM), and plans to initiate a clinical trial in the coming year to evaluate safety and feasibility of extended implantation. While the technology could have future therapeutic potential, near-term clinical investigations are expected to focus on safety and feasibility, including electrical activity recording, with electrical stimulation therapy remaining a longer-term investigational goal.

This preclinical glioblastoma (GBM) study found that the neurotransmitter GABA acts directly on a type of immune cell called a granulocytic myeloid-derived suppressor cell (gMDSC), altering arginine metabolism and making these cells more effective at suppressing the body's anti-tumor immune response -- but only in females. Using female mouse models of GBM, the researchers showed that blocking GABA receptor signaling reduced this immunosuppressive activity and improved outcomes, while having no effect in males, suggesting that GABA signaling may represent a female-specific immunotherapy target. Although GABA is best known as a neurotransmitter in the brain, this study highlights a previously underappreciated role for GABA signaling in regulating immune cells within the tumor microenvironment.

This sex-specific signaling difference identified in mice was also supported by analyses of multiple independent human datasets and patient tumor samples, substantially increasing confidence that these findings are relevant to human GBM, although it remains unknown whether blocking GABA receptor signaling will improve outcomes for women with GBM.