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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09/03/26
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.
08/17/26
NeOnc Reports Positive Phase II Results for Intranasal Glioma Therapy
The investigational treatment NEO100, a highly purified form of perillyl alcohol, showed encouraging preliminary results in a Phase IIa trial for patients with recurrent or progressive IDH1-mutant grade 3 or 4 gliomas. The NEO100 treatment is self-administered by patients through the nose four times a day, using a nasal mask and nebulizer, with the intranasal delivery intended to help overcome the blood-brain barrier. In the single-arm study, 24 patients received treatment in 28-day cycles; researchers compared the results with historical results for patients receiving treatment for recurrent high-grade glioma.
The study met its primary endpoint, with 49% of patients progression-free at six months (compared with an expected 20% based on the study's historical benchmark). Median overall survival was reported as 26 months, with 87% of patients alive at six months, 61% at one year, and 54% at two years. Five patients were still receiving treatment when the preliminary results were reported, including one who had remained progression-free for 19 months, and the company reported several radiographic responses, including one patient whose enhancing tumor largely resolved after prolonged treatment. NEO100 was reportedly well tolerated, with the company describing no significant toxicity even with prolonged treatment, although the full safety and efficacy data have not yet been published in a peer-reviewed journal.
It is important to note that this was a small, non-randomized trial, and our understanding of the prognosis of recurrent IDH1-mutant tumors continues to evolve (for example, recent research suggests that acquired RAS-MAPK mutations may be associated with shorter survival). Despite these caveats, the results are encouraging for a disease with no approved targeted therapies and very few salvage options in the recurrent setting. The company plans to meet with the FDA to discuss the next steps and a potential registrational trial.
08/17/26
Neurocognitive functioning in long-term survivors of glioblastoma, IDH-wildtype and astrocytoma, IDH-mutant, CNS WHO grade 4: A report from EORTC 1419 (ETERNITY)
The ETERNITY study looked at 185 people who had survived at least 5 years after a diagnosis of glioblastoma, IDH-wildtype, or grade 4 IDH-mutant astrocytoma, with patients surviving an average of about nine years. The researchers found that 78% had some measurable impairment in at least one area of thinking or memory. The most common difficulties involved processing speed, mental flexibility, and finding words, while recognition memory was less often affected. Importantly, however, cognitive function was mostly stable over time; the only consistent decline seen across the group was a small worsening in the ability to recall verbal information after a delay. Tumor location mattered; tumors involving the left side of the brain were associated with poorer verbal memory and word fluency, while temporal lobe involvement was particularly associated with memory difficulties. Patients who had experienced multiple tumor recurrences also tended to have worse cognitive flexibility. For patients and families, the findings are encouraging in that surviving many years does not necessarily mean that cognitive abilities will continue to decline, although some lasting difficulties may remain after the tumor and its treatment have affected the brain. The study did not evaluate potential treatments for the cognitive deficits observed, but the authors emphasized that regular neurocognitive monitoring and greater attention to preserving cognitive function should be an important focus of long-term care.
08/17/26
This Deadly Brain Cancer Hijacks Brain Activity to Fuel Its Growth, Study Reveals A new preclinical study from Stanford has added to the growing understanding of how gliomas leverage normal brain activity for growth. Researchers found that NLGN3, a protein made by normal neurons, interacts with the CSPG4 protein found on glioma cells, activating a mechanosensing pathway involving PIEZO1 that ultimately promotes tumor growth. This discovery broadens potential treatment targets beyond NLGN3 itself, possibly to CSPG4; however, the major challenge is that this pathway also appears to help maintain normal oligodendrocyte precursor cells, so blocking it could potentially interfere with healthy brain function. Nevertheless, this work provides important new insights into how gliomas exploit neuronal activity and could help researchers find ways to disrupt this process without harming normal neuron-glial signaling the brain needs.
08/10/26
Webinar this week!
Our next webinar is this Thursday, August 13th at 7pm ET. The topic is "Multi-antigen T-cell therapy in pediatric brain tumors" with Dr. Eugene Hwang. To join, visit virtualtrials.org/webinar.
08/10/26
Ultra-hypofractionated versus conventional chemoradiation for newly diagnosed glioblastoma: Survival and toxicity results of a multicenter randomized trial
A randomized phase III trial from the Netherlands compared an ultra-short radiation schedule (6 treatments of 6 Gy over 2 weeks) with standard radiation for newly diagnosed glioblastoma (30 treatments of 2 Gy over 6 weeks). Both groups received concurrent temozolomide followed by adjuvant temozolomide.
The study enrolled only 135 of the planned 474 patients due to slow recruitment. Even so, the results were concerning. Median overall survival was 13 months with the 2-week schedule versus 21 months with the standard schedule. The 6 × 6 Gy regimen also produced more radiation necrosis or pseudoprogression (47.8% vs 16.2%), and patients receiving the ultra-short treatment were more likely to require dexamethasone later.
It's worth noting that these results do not mean that all shortened radiation schedules are inferior. The Perry regimen, for example, uses 40 Gy in 15 treatments over 3 weeks and is an established option for selected older patients who may not be ideal candidates for the standard 6-week course. However, this study suggests that compressing radiation into just 6 very high-dose treatments is not advised for the general newly diagnosed GBM population.
08/10/26
Butterfly gliomas: to biopsy or to ablate - a longitudinal cohort study
A recent University of Miami study looked at 44 patients with butterfly high-grade gliomas (tumors that cross the corpus callosum and involve both sides of the brain). These tumors can be difficult to safely remove with resection surgery.
In the study, 29 patients received laser interstitial thermal therapy (LITT) and 15 had biopsy alone. Median overall survival was 14.9 months with LITT versus 4.9 months with biopsy, and median progression-free survival was 4.7 verses 2.5 months. The researchers reported that larger tumors were harder to ablate completely with LITT, but surprisingly, the amount of tumor ablated or left behind was not associated with survival. Patients whose functional status remained stable or improved after LITT tended to live longer.
While this study was not a comparison between LITT versus conventional resection surgery, it is worth noting the 14.9-month survival with LITT falls within the range typically reported in previous studies of open resection for butterfly gliomas (roughly 7-15 months).
While this was a small retrospective study, the results support further study of LITT as a minimally invasive option for selected patients with butterfly gliomas.
08/10/26
Distinguishing true progression from treatment effects in glioblastoma: a practical, evidence-graded imaging framework for the multidisciplinary team
One of the biggest challenges after GBM treatment is figuring out whether a new or growing area on an MRI is real tumor growth or treatment effect such as pseudoprogression or radiation necrosis. Conventional MRI cannot reliably distinguish the two in a large portion of cases, often estimated at 30-40%.
Researchers assessed the literature to see how this problem is being addressed, including implementation of the RANO 2.0 guidelines. RANO (Response Assessment in Neuro-Oncology) is an international group that develops standardized guidelines for interpreting brain tumor scans, and its RANO 2.0 guidelines were published in 2023. Based on their review, the authors propose a tiered approach for distinguishing tumor growth from treatment effect: starting with RANO 2.0 MRI assessment, then move to advanced/perfusion MRI, then to amino-acid PET, then to biopsy when necessary. They emphasize that this is a potential framework, not yet a validated recommendation.
For patients, the practical takeaway is that when an MRI is unclear, there are increasingly more tools available to help determine what is really happening, including perfusion and amino-acid PET (and also fractional tumor burden mapping, which we've covered previously), but none can yet guarantee a definitive answer.
08/10/26
An Unexpected Metabolite Reveals a New Weakness in Ependymomas
Researchers at the University of Michigan found that ZFTA-RELA fusion ependymoma (which make up most supratentorial ependymomas) tumor cells produce unusually high levels of itaconate, a substance normally made by immune cells. The tumor appears to use itaconate to help keep the ZFTA-RELA cancer-driving gene switched on, creating a cycle that helps the tumor grow. The researchers also found that the tumor changes the way it uses glutamine, a nutrient that helps provide the building blocks needed to make itaconate.
These discoveries have helped identify potential treatment targets. In mouse models, blocking either glutamine metabolism, the enzyme ACOD1 that makes itaconate, or related signaling pathways slowed tumor growth. Combining these treatments worked even better.
While this research is still preclinical, the University of Michigan team is now working with the Pediatric Neuro-Oncology Consortium (PNOC) to develop a clinical trial based on this research. We will keep an eye out and hope to see the trial open soon!
08/03/26
5th Annual BrainStorm Summit
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.
07/20/26
Musella Foundation's Brain Tumor Guide 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.
07/20/26
Musella Foundation Copayment Program now closed
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!
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