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.

Our partners at Pinpoint Patient Recruiting, a market research recruitment company, are searching for people who have been diagnosed with glioblastoma (GBM), or their care partner, to participate in an online survey about their experiences.  The objective of the survey is to learn more about the experiences of people living with GBM and their opinions on treatments and care. Qualified participants who complete a 30-minute online survey will be paid $75 in appreciation of their input and time. 

You may qualify to complete the survey if you, or the person you care for,  are over the age of 22 and: 
      • newly diagnosed or had a recurrence within the last 3 to 15 months
      • are currently receiving or  pursuing treatment for GBM
      • a resident of the United States

Please note all information and responses will remain confidential. To see if you qualify for the study or to get more information, please visit pinpointpatientrecruiting.com/gbm-survey-al or contact Ashley Maggio at ashley.maggio@pinpointpatientrecruiting.com.

Join us tomorrow for an educational webinar on "Phase 2 Study of frontline BPM31510 with Standard of Care in Newly Diagnosed Glioblastoma" with Dr. David Reardon. To join, visit virtualtrials.org/webinar.

The FDA just approved the first FET-PET imaging drug in the U.S. (Pixclara®) for patients with glioma, including adults and children 1 month and older. FET-PET has been studied extensively in Europe and is recommended as an additional diagnostic tool in clinical practice guidelines, including the NCCN Guidelines. It provides information about tumor biology that is different from conventional MRI and may be particularly helpful when a brain MRI is difficult to interpret. Studies of FET-PET have reported sensitivity of approximately 80% or higher for helping distinguish tumor progression from treatment-related changes.

From our friends at the End Brain Cancer Initiative, registration is now open for the Caregivers Self-Care and Tools online educational event on November 6, 2026 at 11am PT. The Keynote Speaker for the event is Dr. Jennifer Nosker, a clinical neuropsychologist and scientist at Brigham and Women's Hospital and Instructor at Harvard Medical School. Dr. Nosker will provide education to caregivers about the different types of cognitive changes that can occur in brain tumor patients, while simultaneously giving practical tools to help navigate these challenges. To register, click here!

A new study of 176 patients with IDH-wildtype glioblastoma (GBM) found evidence of intratumoral hemorrhage on MRI in 105 patients (59.6%). Previous studies have reported much lower rates of 2.3%-29.2%, and the authors caution that differences in MRI techniques and definitions may explain the discrepancy. The authors note their study did not use susceptibility-weighted MRI or confirm hemorrhage pathologically; therefore, 59.6% should not be interpreted as the true percentage of GBMs that hemorrhage.

The researchers found that hemorrhagic tumors had more SETD2 alterations and fewer alterations in PDGFRA, KIT, KDR and PIK3R1, genes involved in tumor growth and blood-vessel signaling. However, none of these differences remained statistically significant after correction for multiple comparisons, making the findings preliminary. Importantly, hemorrhage in GBM was not associated with shorter progression-free or overall survival. The findings suggest that the tendency of some GBMs to bleed may be linked to tumor biology, but larger studies are needed to confirm the molecular associations and determine their clinical significance.

Researchers at MIT have developed an experimental technology called HITMAN (highly-localized electric-field-induced tumor therapy using magnetically actuated nanoantennas), which uses tiny injectable devices to generate localized electric fields that kill cancer cells. The approximately 150-nanometer devices can be activated wirelessly from outside the body using a low-frequency magnetic field. In early preclinical experiments using patient-derived, chemotherapy-resistant glioblastoma cells, HITMAN killed 52.2% of cancer cells, while sparing healthy neurons and astrocytes.   

The researchers then implanted the patient-derived tumors into the brains of mice. HITMAN substantially slowed tumor growth and extended median survival, with no detectable toxicity to surrounding healthy tissue or major organs. The treatment also reduced the ability of tumor cells to form new colonies, suggesting potential to reduce recurrence. The researchers believe the localized electric fields disrupt cancer cells' bioelectric activity and cause protein-folding stress and membrane damage, ultimately leading to cell death. The technology has not yet been tested in humans, but it is an interesting new approach for electrical field therapy. 

Dichloroacetate (DCA) inhibits pyruvate dehydrogenase kinase (PDK), helping shift cancer cells away from the “Warburg effect” (their tendency to rely heavily on glycolysis and produce excess lactate). Earlier preclinical studies provided evidence that DCA may alter GBM metabolism and potentially make tumor cells more vulnerable to cell death.

A new study has now provided additional evidence that DCA reaches recurrent GBMs and produces the intended metabolic changes. In 37 patients undergoing surgery for recurrent GBM, patients received either one week of DCA or no DCA prior to surgery. The resected enhancing and non-enhancing tumor tissue, and serial plasma DNA and lactate levels were analyzed. Patients who received the DCA had reduced markers of PDK activity and tumor-cell proliferation and significantly lowered blood lactate levels. DCA was well tolerated, but the study did not show that DCA improved progression-free survival. The researchers conclude that, although the study did not show a clinical benefit, the metabolic effects warrant further investigation, particularly in combination with other treatments. 

 We recently reported on NEO100, an unusual treatment that delivers highly purified perillyl alcohol directly through the nose, allowing patients to self-administer the drug at home. Earlier results were intriguing, particularly because some of the longest survivors had IDH1-mutant tumors.