Brain Tumor News!
Note: The comments under each article title are the opinion of our president, Al Musella, DPM,
and do not reflect official policy of the Musella Foundation!
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09/28/26
Help needed for research project! 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.
09/28/26
Imvax Glioblastoma Data Backs Move Toward Phase 3 Trial
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.
09/28/26
Scientists find the gene that drives T-cell exhaustion, revealing new strategy to improve immune responses
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.
09/28/26
Divergent medulloblastoma chromatin states disclose KDM2B as a selective dependency
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.
09/28/26
Harvard event on 'The Voice of Glioblastoma' 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.
09/28/26
Share Your Experiences With Glioblastoma - Earn $75
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.
09/14/26
Webinar tomorrow! 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.
09/14/26
FDA Approves Telix's Brain Cancer Imaging Drug Pixclara 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.
09/14/26
Free webinar for caregivers
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!
09/14/26
New molecular insights into intratumoral hemorrhage in IDH-wildtype glioblastoma
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.
09/14/26
Injectable nanodevices could provide effective treatment for drug-resistant glioblastoma
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.
09/14/26
A novel approach to reverse Warburg metabolism in patients with recurrent glioblastoma: A phase II pharmacodynamic study of dichloroacetate
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.
09/03/26
What is MGMT?
One of the most frequently asked questions we get is: “What is MGMT?”
MGMT is one of the most important biomarkers in glioblastoma, but it can be confusing to understand. We created this short animated video to explain what MGMT is, why it matters, and how it can affect treatment decisions.
This is our first time using AI to help create an educational video. The script was carefully reviewed and approved by our team, including Dr. Steven Brem.
We’d really like your feedback! What do you think of this format? Should we make more videos like this? Was it too technical, not technical enough, or just right? Most importantly, were you able to understand it?
And please give us ideas for topics you’d like us to explain in future videos!
09/03/26
NEO100-01 Meets Primary End Point, Supporting Further Development in IDH1-Mutant Glioma 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.
These new Phase 2a results strengthen that story. In 24 patients with recurrent or progressive IDH1-mutant grade 3 or 4 glioma, the 6-month progression-free survival rate was 48.9%—more than double the study's prespecified 20% benchmark—and median overall survival was an impressive 26.1 months. The treatment was also reported to be generally well tolerated, with no major toxicities.
There are important limitations. This was a small, single-arm study, so the results need to be confirmed in a larger trial with an appropriate control group. The objective response rate was only 8.3%, reminding us that prolonged disease control does not necessarily show up as dramatic tumor shrinkage on MRI.
Still, a median survival of more than two years in patients with recurrent high-grade IDH1-mutant glioma deserves attention. Perhaps just as important, NEO100 appears relatively easy for patients to use compared with many experimental brain tumor treatments. The company now plans to meet with the FDA to discuss a possible registrational pathway.
We will be watching closely. If these results hold up in a larger study, NEO100 could become an important new option for this molecularly defined group of brain tumor patients.
08/27/26
New oral nanomedicine boosts effectiveness of cancer immunotherapies Can a Gut-Derived Compound Make Immunotherapy Work Better?
One of the biggest frustrations with cancer immunotherapy is that, while treatments such as checkpoint inhibitors can produce dramatic responses in some patients, they do very little for many others. Researchers at the University of Michigan may have found an interesting way to improve those odds.
They developed an oral nanomedicine based on 3,4-dihydroxybenzoic acid (DHB), a natural compound produced by gut bacteria when dietary fiber is broken down. DHB appears to help T cells maintain a more durable, “memory-like” state instead of becoming exhausted and losing their ability to attack cancer.
Because DHB itself is poorly absorbed and quickly eliminated, the researchers converted it into a prodrug and packaged it in a nanoemulsion designed to improve delivery. In mouse models of melanoma, colorectal cancer and breast cancer, combining the oral treatment with checkpoint inhibitors eradicated tumors and generated immune memory that protected against recurrence. The treatment also improved the activity of CAR T-cell therapy.
For the brain tumor community, the concept is particularly interesting. Glioblastoma has historically responded poorly to checkpoint inhibitors, in part because its immune environment suppresses T-cell activity. Anything capable of keeping T cells functional longer could potentially help immunotherapies work better.
However, there is an important caveat: this research has so far been demonstrated in mice, not patients, and glioblastoma was not one of the tumor models reported in this study. We have seen many promising immunotherapy approaches cure tumors in mice only to disappoint in human trials.
Still, I think this is an intriguing avenue of research. Instead of inventing an entirely new immune therapy, researchers may be able to make existing treatments work better by changing the metabolic environment of the immune cells themselves. If this approach translates to humans, combinations with checkpoint inhibitors, vaccines, CAR T cells or other immune therapies could eventually be worth exploring in brain tumors.
As always, the next important step is human clinical testing.
08/23/26
Battleship of Experiments: The Fight Against Brain Cancer This is an interesting example of how advances against glioblastoma may come from **combining treatments rather than continually searching for a single “magic bullet.”** Researchers are studying whether extracellular vesicles – tiny particles that can cross the blood-brain barrier – can make glioblastoma cells more vulnerable to CAR T-cell therapy. They are using mathematical modeling and sophisticated image analysis to understand exactly how the two interact.
I particularly like the multidisciplinary nature of this project. Biology, immunology, physics, mathematics and computer programming are all being brought together to attack the same problem. The researchers are even developing software to analyze hundreds of microscope images and quantify how extracellular vesicles are being absorbed by tumor cells and how that affects CAR T-cell killing.
Of course, this is still early laboratory research and is a long way from proving that the combination will help patients. But glioblastoma has defeated many promising single treatments. Finding ways to intelligently combine therapies – and using computational tools to determine which combinations actually work synergistically – may be one of our best paths toward meaningful progress.
08/21/26
Novel Radiation Strategy Redefines a New Treatment Paradigm for Glioblastoma
This is an interesting approach that makes a lot of sense. After glioblastoma surgery, we usually wait several weeks for the surgical wound to heal before starting radiation and chemotherapy. Unfortunately, glioblastoma does not wait.
GammaTile consists of small absorbable tiles containing radioactive cesium-131 seeds that are placed directly into the tumor cavity at the time of surgery. This allows radiation to begin immediately, targeting residual tumor cells while limiting radiation exposure to the surrounding brain. Patients then go on to receive a shortened course of traditional radiation along with standard chemotherapy.
In this 67-patient Phase I study, only 6% of patients experienced rapid tumor regrowth before beginning the next phase of treatment, compared with historical rates reported as 50–70%. That is certainly encouraging.
However, this was not a randomized trial, so we cannot yet say that GammaTile improves survival or should become the new standard of care for newly diagnosed GBM. A Phase III randomized trial called BRIDGES is now underway to answer that question.
I like the concept. Instead of giving residual glioblastoma cells several weeks to recover and grow after surgery, start attacking them immediately. Now we need the randomized trial to tell us whether that translates into longer survival.
Disclaimer: GT Medical is a proud sponsor of the Musella Foundation!
08/21/26
Enrollment opens for Adaptin Bio’s Phase I trial of glioblastoma therapy
This is an interesting new approach that is now making the important jump from laboratory studies into its **first human trial**.
Adaptin Bio has opened enrollment at Duke University for a Phase I trial of **APTN-101**, a treatment designed for glioblastomas that express **EGFRvIII**. APTN-101 is what the company calls a Brain Bispecific T-cell Engager, or **BRiTE**. The idea is to use manipulated T cells not only to attack the tumor, but also to help carry the therapeutic agent across the blood-brain barrier and directly to glioblastoma cells.
I find this particularly interesting because it tries to address two major problems in glioblastoma at the same time: **getting an effective treatment into the brain and directing the immune system specifically against the tumor.** The treatment targets EGFRvIII, a tumor-specific alteration found in a subset of glioblastomas, which theoretically provides a way to attack tumor cells while sparing normal cells.
Preclinical studies reportedly showed activity against patient-derived glioma cells and encouraging results in animal models, but we have to remember that many GBM treatments that look impressive in the laboratory ultimately fail in patients. This Phase I study is primarily designed to determine **safety and the appropriate dose**, not to prove that the treatment extends survival. It plans to enroll up to 15 adults with EGFRvIII-positive malignant glioma.
Still, this is exactly the type of trial I like to see: a scientifically interesting idea has moved beyond the laboratory and is finally being tested in patients. **Now we get to find out whether the concept actually works in the human brain.**
08/21/26
Screening newborns for cancer risk
This is a fascinating idea, particularly for brain tumors. Researchers looked at newborn blood samples from 1,948 children who later developed a solid tumor or brain tumor by age 8 and found that about 7% carried a detectable mutation in one of 11 cancer-predisposition genes. For some brain tumors the numbers were much higher: mutations were found in about 30% of children who developed choroid plexus carcinoma, 17% with pineoblastoma and 11% with medulloblastoma.
Knowing about these mutations at birth could allow doctors to monitor high-risk children closely and hopefully find a tumor when it is very small, potentially improving survival and allowing less-toxic treatment.
But there is an important downside that needs to be considered. These cancers are extremely rare. The researchers estimate that only about 1 out of every 27,000 newborns would go on to develop a cancer by age 8 that could have been predicted by this 11-gene screening panel.
That does not necessarily mean 26,999 children would undergo MRIs and other intensive screening to help one child. The initial genetic test would be performed on the blood spot already routinely collected from newborns, and only children found to carry a significant mutation would be referred for additional surveillance. Importantly, these mutations appear to be quite rare in healthy newborns.
Still, for a child who tests positive, surveillance can become a significant burden. Depending on the particular gene, it can involve repeated physician visits, blood tests, ultrasounds, eye examinations and imaging such as MRI—sometimes beginning in infancy and continuing for years. There is also the possibility of anxiety for the family, overdiagnosis and procedures prompted by findings that ultimately prove harmless. The study authors themselves acknowledge these concerns.
So I think the concept is promising, especially for syndromes where the risk of developing cancer is very high and we already know that early detection makes a difference. But before adding cancer genes to routine newborn screening, we need to know something this study cannot yet tell us: How many children need years of additional surveillance to prevent one cancer death or serious disability?
Finding a genetic risk is only valuable if acting on that information does more good than harm.
08/21/26
BioArctic and Mesenkia Partner to Develop Brain-Penetrant Antibody for Glioblastoma
This is an interesting new approach, but it is important to understand that it is still at the **preclinical research stage**—there is not yet a drug being tested in glioblastoma patients.
BioArctic and Mesenkia are combining two technologies. Mesenkia has developed an antibody that targets **HVEM**, a protein found on some glioblastoma cells, including tumor stem cells that may play an important role in treatment resistance and recurrence. BioArctic is adding its **BrainTransporter** technology, which is designed to carry antibodies across the blood-brain barrier.
That second part may be the most interesting. One of the major reasons many promising drugs fail in brain tumors is simply that not enough drug reaches tumor cells scattered throughout the brain. In preclinical studies, BioArctic reported that its BrainTransporter increased antibody exposure in the brain by as much as **70-fold**.
If this technology really can safely deliver large antibody drugs throughout the brain, its importance could eventually extend well beyond this particular antibody. It might provide a way to revisit other promising treatments that previously could not reach adequate concentrations in the brain.
However, we are a long way from knowing whether this will help patients. The companies still have to create the combined drug candidate and test it preclinically before deciding whether to move into human trials.
I am encouraged by the concept, particularly because it attacks **two problems at once—finding an important target on resistant glioblastoma cells and figuring out how to get the treatment to those cells.** Now we have to see if it works.
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