The investigational drug tinostamustine (EDO-S101) will soon be added as a treatment arm in the Phase 2/3 GBM AGILE trial—a global, adaptive study designed to accelerate development of new therapies for glioblastoma. The GBM AGILE trial tests multiple therapies simultaneously against a shared control group and provides a potential pathway to FDA approval. Tinostamustine is a first-in-class compound that combines bifunctional alkylating activity with pan-HDAC inhibition, two mechanisms that may work together to attack glioblastoma cells more effectively. MD Anderson recently completed a small Phase 1 study of the drug in newly diagnosed, MGMT-unmethylated glioblastoma, but the results have not yet been published. In GBM AGILE, the drug will be evaluated in both newly diagnosed and recurrent glioblastoma patients. Activation of this trial arm is expected after regulatory submissions are approved, and we will be closely following for further updates.

A new study from researchers at Sylvester Comprehensive Cancer Center in Miami has shown that the spatial organization of glioblastoma (GBM) cells may play a key role in treatment resistance and clinical outcomes.

Using single-cell spatial transcriptomics, the researchers showed that GBM cells organized in homotypic clusters maintain stable identities, while dispersed cells exhibit increased plasticity, shifting to alternative phenotypes and altering their surrounding microenvironment. This dispersed, glycolitic-plurimetabolic cell state was associated with significantly shorter survival.

These findings raise the possibility that standard treatments—while essential—could inadvertently contribute to dispersal of GBM cells, potentially promoting a more therapy-resistant phenotype. While this hypothesis requires further validation, it is an important consideration for future therapy development.

Please join us on Thursday, September 18 at 7pm ET for "BRAF-Targeted Therapies for Brain Tumors" with Dr. Karisa Schreck.  

A new study from MD Anderson Cancer Center reports promising outcomes using GammaTile (cesium-131 collagen tile) brachytherapy for brain metastases recurring after prior stereotactic radiosurgery (SRS). Among 31 patients with brain metastases, most commonly from breast (37%) or non-small cell lung cancer (26%), all underwent surgical resection with GammaTile placement. Gross total resection was achieved in 84% of cases. At a median follow-up of 11.8 months, local failure occurred in 13% overall and just 6.4% when complete resection was achieved. One-year overall survival was 65.8%, with low toxicity—only 8% developed grade 2+ radiation necrosis. Subtotal resection, tumor volume, multiple prior radiation courses, and dural contact were associated with higher failure risk. These results support GammaTile as a potential effective, low-toxicity salvage option for recurrent brain metastases.

The Musella Foundation is honored to welcome Dr. Steven Brem as its Chief Scientific Advisor. Dr. Brem is a world-renowned neurosurgeon, educator, and innovator in the field of brain tumor research and care. He currently serves as Professor and Director of Medical Student Education in the Department of Neurosurgery, Medical Director of the Center for Precision Surgery, and Senior Member of the Penn Brain Tumor Center. For the full press release, please visit HERE. 

Mark your calendars! Our next webinar is coming up on Thursday, September 18 at 7pm ET with Dr. Karisa Schreck on "BRAF-Targeted Therapies for Brain Tumors"!

The 6th Annual Mark Salkowski 5K Run/Walk will be held on Sunday, September 14, 2025, at the Falling Branch Brewery in Street, MD. All proceeds benefit brain cancer research conducted by the Musella Foundation. Mark was a loving father, husband, brother, and friend to many. He sadly passed away from glioblastoma, a rare and deadly form of brain cancer, on December 28, 2015. This event is to honor Mark and raise funds in his name to support the fight against brain cancer. Registration begins at 8:00 with the run/walk starting at 9:00. After the event there will be a silent auction, music, and food available and beer for those over 21 years of age. Come out and join us for a great cause and to honor a great man. Questions can be directed to Neil Salkowski at neilsal@aol.com. More information is available and to register or donate, please go to: Musella Foundation: 6th Annual Mark Salkowski Memorial 5K and 1K Fun Run. 

This single-institution retrospective study reviewed outcomes in 63 patients who underwent surgery for brain metastases, comparing those who received carmustine (BCNU) wafers at the time of resection to those who did not. Lung cancer was the most common primary cancer in the cohort. Progression-free survival (PFS) was significantly improved in the BCNU group versus the surgery-only group. Median overall survival from the time of surgery was 10 months with BCNU versus 7 months without, a difference that did not reach statistical significance. This is the largest modern, two-arm study of BCNU wafers in brain metastases, and it supports their potential to improve local control when used alongside surgery.  

The Pediatric Neuro-Oncology Consortium (PNOC), a global network of over 300 researchers across 43 institutions, has launched the Lose No Child campaign—a $50 million initiative to fast-track safer, more effective treatments for children with brain cancer. The PNOC Foundation aims to cut development timelines in half by funding clinical trials, expanding access to innovative therapies, and advancing AI-driven research. Learn more at: www.losenochild.org.

The 5th International Gliomatosis Cerebri (GC) Conference will be held in London on September 17-19. This conference builds on the success of earlier meetings in Paris, Bethesda, Barcelona and New York City and aims to bring together clinicians, researchers and families to hear about the latest research in GC and discuss ways to make progress for this disease. The event is invitation only; if interested in attending, please contact Chris Jones, PhD, Institute of Cancer Research at chris.jones@icr.ac.uk.  

Join us for "Integrative Medicine for Patients with Primary Brain Tumors" with Dr. Nicholas Butowski on Wednesday, August 27 at 8:30 ET/5:30 PT. Click on Brain Tumor Webinars to attend!

Mark your calendars for Wednesday, August 27, 2025 at 8:30 ET/5:30 PT! We will be having a fantastic webinar with Dr. Nicholas Butowski on "Integrative Medicine for Patients with Primary Brain Tumors" (This webinar was rescheduled from last month.) 

Good news from the Phase 1 ReSPECT-LM trial evaluating an investigational radiotherapeutic (REYOBIQ™) in patients with leptomeningeal metastases (LM) - a rare, aggressive complication in advanced cancers. Among 29 patients treated in escalating dose cohorts, there was a 76% radiographic response rate and 87% clinical response rate. A strong biological effect was also observed; patients had up to 100% reduction in tumor cells found in the cerebrospinal fluid, and five of seven patients with major tumor cell reductions survived at least one year, which is well above the typical 2-6 month median survival for LM.

Across the first four dosing cohorts (20 patients),median survival was 9 months, which is an improvement over historical outcomes. Side effects were mostly mild (grade 1–2), and only two patients experienced serious dose-limiting toxicity (grade 4 cytopenia) at the highest dose levels. Based on this trial, the team has identified a recommended dose for a Phase 2 trial. These early results suggest REYOBIQ has the potential to become a much-needed targeted treatment option for LM.

A recently published Phase 1/2a study evaluated Lisavanbulin, an oral microtubule-targeting agent, in patients with recurrent glioblastoma (GBM) and other high-grade gliomas (HGGs). In the Phase 1 trial, 28 patients received daily Lisavanbulin at doses up to 30 mg. Among them, one patient had a confirmed complete response (CR) and one had a confirmed partial response (PR) with 94% tumor shrinkage; both remained on therapy for over four years. An additional seven patients had stable disease, yielding a disease control rate (DCR) of 32.1%. However, median progression-free survival (PFS) was just 1.6 months, and only 11.2% of patients remained event-free at 12 months.

In the Phase 2a expansion, 13 patients with recurrent GBM were pre-selected based on EB1 protein expression, a potential biomarker of response. Of nine with measurable disease, one patient had a durable PR and four had stable disease, for a DCR of 55.6%. Overall, the 12-month event-free survival was 32.5%, and 12-month overall survival was 55.6%, but the study did not meet its pre-specified efficacy threshold and was closed to further recruitment. Follow-up studies are planned to investigate why a small subset of patients had such exceptional and durable responses, with a focus on refining EB1 as a predictive biomarker to better identify likely responders.

The Social Security Administration has added Grade III meningioma to its Compassionate Allowances List (CAL), which will make it faster and easier for patients with this type of brain tumor to access disability benefits. Normally, applying for Social Security Disability Insurance (SSDI) can take months or even years, but inclusion on the CAL means that applications will be fast-tracked with minimal documentation and quicker decisions. This is an important advancement for patients and families facing the challenges of high-grade meningioma, helping them get financial support more efficiently.

From our friends at the Brain Injury Association of New York State (BIANYS), their March On for Brain Injury Walk will take place at Eisenhower Park in Scarlett Oak, Long Island on September 13, 2025! For more information and to register, click here.

This trial compared a combination of two immune checkpoint inhibitors (ICIs), ipilimumab plus nivolumab, versus standard temozolomide in patients with newly diagnosed MGMT-unmethylated glioblastoma (GBM). We had high hopes for this one, but unfortunately, preliminary results showed there was no improvement in progression-free survival (7.7 months with the combination vs. 8.5 months with TMZ). Early overall survival data also showed no difference, so the trial has been stopped. Follow-up and biomarker analyses are ongoing to explore potential subgroups that may benefit from this immunotherapy combination.

So far, studies using ICIs in GBM generally have not had great results. However, we're hopeful they will still have a role to play, perhaps with a better understanding of molecular feature selection and/or when given in the neoadjuvant setting. We are also very hopeful for the Phase 3 EF-41 trial, which is testing the combination of the ICI pembrolizumab (Keytruda) together with tumor treating fields (Optune Gio); this combination acheived significantly improved median survival (24.8 vs. 14.6 months) in the Phase 2 "2-THE-TOP" trial for newly diagnosed GBM, especially for patients who only had biopsy and were not eligible for resection (31.6 vs. 18.8 months).

 This is the drug that used to be called Onc-201.  It finally got approved as the first drug ever approved for diffuse midline glioma!
The Musella Foundation supported the development of this drug from the start!

 We have run out of funding, and the copay program is now closed. We will reopen again when we are able to raise more funds. Of course, we will continue to pay claims for those that have an active grant!