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.

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 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.

Another study from researchers at Northwestern has also identified a mechanism by which glioblastoma suppresses the immune system. They found that tumor cells express a protein called podoplanin (PDPN), which binds to the receptor CLEC5A on tumor-associated macrophages in hypoxic, necrotic regions of the tumor. This interaction activates the Syk-JAK-STAT3 signaling pathway, driving macrophages into an immunosuppressive state that helps the tumor evade immune attack and is associated with worse patient outcomes. In mouse models, blocking CLEC5A or inhibiting Syk signaling reduced immune suppression, slowed tumor growth, and extended survival.

Importantly, some Syk inhibitors are already FDA-approved for other diseases, raising the possibility of faster clinical translation. The authors also suggest that reversing this macrophage-driven immunosuppression may not be sufficient on its own, and that future strategies may need to combine this approach with therapies that enhance cytotoxic T-cell activity, such as checkpoint inhibitors, in order to improve immune recognition and tumor targeting in GBM.

The Phase 3 TRIDENT trial enrolled 981 newly diagnosed glioblastoma (GBM) patients to evaluate whether starting Tumor Treating Fields (TTFields) during radiation and temozolomide treatment could improve survival compared with the current approach of starting TTFields after chemoradiation is completed.

The trial did not meet its primary endpoint, with median overall survival of 17.7 months in the early-start group versus 17.5 months in the standard group. However, these results should not be interpreted as a negative study for TTFields itself. Both groups received TTFields; the question being tested was whether starting treatment earlier would provide additional benefit.

One encouraging finding was the long-term survival tail in both groups. Three-year survival rates were 22.5% in the early-start arm and 18.4% in the standard-start arm, which compares favorably with historical outcomes for newly diagnosed GBM. 

While earlier initiation did not significantly improve overall survival in the overall study population, additional analyses may identify patient subgroups that derive greater benefit from earlier treatment. Novocure has not yet released detailed compliance (usage) data, which may provide additional insights when the full results are presented at the ASTRO 2026 Annual Meeting.

Do you know a college student (ages 18–23) whose parent has been diagnosed with or passed away from a primary brain tumor? Our friends at the George Bartol Memorial Scholarship Fund are once again awarding scholarships to students across the United States impacted by a parent’s brain cancer diagnosis.

Eligibility:
- Full-time student at a 2- or 4-year college/university
-Minimum 2.5 GPA
-Ages 18–23

Applications are due by October 1, 2026! To request an application, please message the George Bartol Memorial Scholarship Fund on Facebook! 

 As a friendly reminder, brain tumor patients and caregivers can receive free one-on-one navigation support through the Brain Cancer Support & Solutions Alliance (BCSSA), a program jointly funded by Cancer Commons, Head for the Cure, and the Musella Foundation. You can enroll at diagnosis, recurrence, or anytime in between. We know how difficult the brain tumor journey can be, and we're here to help. To learn more or register, click HERE. 

Researchers in Ireland have reported the first preclinical study of an mRNA vaccine designed specifically to treat neuroblastoma, one of the deadliest childhood cancers.

This vaccine is designed to target Glypican-2 (GPC2), a protein found at high levels on many neuroblastoma cells and also expressed in medulloblastoma and certain other high-grade gliomas. Instead of using traditional lipid nanoparticles for the vaccine, they employed tiny self-assembling peptide nanoparticles called RALA to deliver the mRNA instructions to the immune system.

The vaccine successfully stimulated a strong anti-tumor immune response in lab models, increasing production of several immune signaling molecules, including IFN-γ, IL-2, and TNF-α. In mice with aggressive MYCN-amplified neuroblastoma, the vaccine delayed tumor growth by roughly 10-11 days and reduced tumor volume by ~70% compared with untreated animals.

These results are preclinical, and significant additional work will be needed before human trials can test the safety and efficacy of this vaccine. Nevertheless, we will keep an eye on this promising research.

This Phase 1 GT-20 trial tested a personalized DNA neoantigen vaccine in 9 patients with newly diagnosed MGMT-unmethylated glioblastoma (GBM). Unlike other neoantigen vaccine approaches, GT-20 sequenced multiple regions of each tumor rather than just one and incorporated up to 40 patient-specific neoantigens into each vaccine.

The vaccine was generally well tolerated and generated measurable immune responses in six of seven evaluable patients, with evidence that vaccine-induced T cells entered the tumor microenvironment. The study was not designed to evaluate efficacy, but investigators reported a median overall survival of 16.3 months and a two-year survival rate of 33%. Patients receiving dexamethasone had weaker immune responses, consistent with findings from other immunotherapy studies.

While several neoantigen vaccine programs have successfully generated anti-tumor immune responses in GBM, the key question remains whether those responses can translate into meaningful clinical benefit, either alone or in combination with other therapies.

We’re excited to see these Phase 1 study results now published in Neuro-Oncology Advances.

The drug appeared safe and generally well tolerated, and the median overall survival was about 16 months, which compares favorably to historical outcomes in recurrent glioblastoma. While this is a small, non-randomized trial of only 22 analyzed patients, thes results are encouraging for a Phase 1 study in this setting.

Most patients still followed the expected course of progression over time, but five of the 22 included patients are still alive. Four of those are beyond 18 months, and two of them are at 31 and 39 months since starting treatment with gallium maltolate.

We helped fund an expanded access program for oral gallium maltolate, and we’re encouraged to see it is now expected to open to about 20 additional patients, giving more families access while the research continues.

This signal concerning proton pump inhibitors (PPIs) first emerged from analyses of real-world registry data from the xCures database, which suggested a potential link between PPI use and shorter survival in glioblastoma (GBM) patients. It is a good example of how patient registries and real-world evidence can help identify clinically relevant signals early, often years before they are evaluated or confirmed in larger randomized clinical trial datasets. Now, the association has been independently validated in a large analysis of nearly 3,000 patients from five randomized GBM clinical trials.

The recent study found that patients taking certain PPIs, including omeprazole and pantoprazole, had shorter progression-free and overall survival, even after adjusting for key factors such as age, MGMT status, extent of surgery, and steroid use. Other stomach-protecting medications, such as famotidine (Pepcid), did not show the same association, suggesting the effect may not simply be related to acid suppression.

This does not prove that PPIs cause worse outcomes, but the consistency of the signal across datasets adds to the concern. Since many GBM patients are placed on PPIs automatically when steroids are prescribed, it is reasonable to periodically reassess whether a PPI is truly needed or whether an alternative agent could be used instead.

As always, patients should not stop prescribed medications without discussing it with their medical team.