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!

The Musella Foundation Copayment Assistance Program is running low on funding and will close to new and renewal applications soon. Of course, we will continue to pay claims for those already approved - just will not be able to accept new applications. So if you have been thinking of applying, go to braintumorcopays.org and follow the directions to send in an application!

This was an interesting study done in Taiwan where they used a rotating chemotherapy approach called BAC (bevacizumab alternating chemotherapy) in adults with recurrent high-grade glioma, including glioblastoma. The BAC regimen included 2 cycles of etoposide + carboplatin, followed by 1 cycle of cyclophosphamide + vinblastine, with bevacizumab every 4 weeks. In the patients with grade 4 gliomas, those treated with BAC lived a median of 29 months compared to 19 months with bevacizumab alone, and had 16 months of survival after recurrence versus 10 months. The benefit was especially strong in patients with MGMT-methylated tumors, who lived up to 33 months. While this was a relatively small trial from a single institution, BAC may be an option to consider for recurrent high grade glioma patients who aren’t eligible for clinical trials.

A new study from Dana-Farber and the Broad Institute, published in Nature Communications, found that 8.9% of pediatric gliomas -- especially low-grade gliomas -- have FGFR gene alterations. An initial retrospective look shows that these tumors may respond to existing FGFR inhibitors. While more trials are needed, this discovery could lead to new precision therapy options for children with pediatric low grade gliomas.

We’re excited to share that Unsha Bakker, Nurse Navigator for the Brain Cancer Support and Solutions Alliance (BCSSA), was recently featured on Head for the Cure's Rare Enough podcast. The BCSSA is a collaborative initiative between the Musella Foundation, Head for the Cure, and Cancer Commons, offering free personalized guidance to brain tumor patients and families. In this episode, Unsha discusses her vital role with the BCSSA, as well as her journey into nursing, the barriers underserved communities face in accessing care, and the human side of nursing. To listen, click here. 

This thoughtful and wide-ranging Substack article by Dr. Wafik El-Deiry is worth a read. Reflecting on a recent interview with Senator Bill Cassidy, Dr. Patrick Soon-Shiong and Mark Halperin, Dr. El-Deiry covers topics including the promise and limitations of immunotherapy, the evolution of precision oncology, access disparities in rural America, and the long road of drug development. He gives some great insights on where cancer care is headed—and what still stands in the way.

The annual Tumor Takedown fundraiser is taking place again in Cape Coral, Florida this Saturday, July 19th at 5pm ET. This event is put on by Zachary and Morgan, brain tumor survivors and thrivers, to raise local awareness for brain cancer. All proceeds go to the Musella Foundation for brain tumor research! For more info, click here. 

We gave a grant last year for a preclinical project exploring heat-sensitive liposomes (Thermodox®) combined with laser interstitial thermal therapy (LITT). This year, we awarded a $50,000 grant to Dr. Brice Martin to at Weill Cornell to investigate low-intensity focused ultrasound (LIFU) + Thermadox® for diffuse midline glioma (DMG). The special heat-activated chemotherapy formulation of Thermodox® helps bypass the blood brain barrier and deliver targeted therapy directly to the tumor site. This approach could significantly improve the safety and effectiveness of chemotherapy, and our support will help build the preclinical groundwork needed to move this technology into human trials for GBM and DMG.

This is an interesting drug combination strategy aimed at enhancing standard of care for glioblastoma (GBM) by targeting multiple pathways involved in tumor growth. Like the earlier CUSP9v3 protocol, AVRO proposed repurposing well-tolerated, FDA-approved non-oncology drugs based on preclinical evidence of GBM inhibition. The article mentions potential interaction between levetiracetam (Keppra) and vortioxetine/olanzapine and thus mentions lacosamide (Vimpat) may be a preferable seizure prophylaxis for patients under the AVRO regime. 

Magnetic resonance spectroscopic imaging (MRSI) has been explored for decades in brain tumor imaging, but clinical adoption has been limited due to long scan times and low signal clarity. Researchers at the University of Illinois Urbana-Champaign recently reported a novel MRSI technique that combines ultrafast data acquisition with machine learning-powered image reconstruction. Their method reduces whole-brain scan time to just 12.5 minutes while significantly enhancing image resolution and metabolite signal quality—making metabolic imaging much more feasible in clinical workflows. Other leading centers, including Cornell, Emory, and the University of Miami, are also advancing MRSI technology, and we hope to see this translate to improved routine clinical care for glioma patients.

The University Medical Center in Nashville, Tennessee is hosting a paid virtual discussion for caregivers of individuals that have been diagnosed with glioblastoma (GBM). The purpose of the meeting is to specifically focus on the opinions caregivers have on a clinical trial to offer a new surgical intervention in order to improve quality of life after diagnosis. It will be a one-time, 2-hour virtual meeting via Zoom, and participants will be compensated with a $66 e-gift card. To fill out an interest survey for the discussion, click here!

Our brain tumor treatment copay assistance program is now open to new (and renewal) patients again. This program can help cover: Optune, Avastin, Temodar, and Gleostine and their generics. If you think you may need help, apply ASAP as the program might not be open too long. Go to braintumorcopays.org for details and to apply!