Our brain tumor copayment assistance program is now open to new (and renewal) patients. This program can help cover the copay costs for: Optune, Avastin, Temodar, and Gleostine, as well as their generics. If you think you may need help, apply ASAP as the program often closes quickly. Go to braintumorcopays.org for details and to apply!

Recently published results from a multi-center phase 1/2 trial suggest that MRI-guided microbubble focused-ultrasound (MB-FUS) may improve outcomes when added to standard chemotherapy for newly diagnosed high-grade glioma. The study enrolled 34 newly diagnosed high-grade glioma patients between 2018 and 2022 (pre-2021 WHO reclassifications). Most of the tumors were IDH-wildtype, with a mix of MGMT-methylated and unmethylated cases. 

After surgery and standard chemoradiation, patients received monthly outpatient MB-FUS treatments given once at the start of each temozolomide cycle to temporarily open the blood–brain barrier in large, contoured regions. A median of four MB-FUS cycles were completed per patient (range 1-6), with some missed cycles due to COVID-19 disruptions. 

Despite the small size of the study, patients treated with MB-FUS and temozolomide had significantly longer progression-free survival (13.5 months) and overall survival (31.3 months) compared with a carefully matched external control group treated with temozolomide alone, and the survival benefit remained consistent across multiple sensitivity analyses designed to account for bias. Side effects related to the focused ultrasound were mostly mild, with no unexpected safety concerns. These early results provide strong support for larger, randomized trials to test this treatment strategy.

The STELLAR trial was a large, randomized multi-center study testing eflornithine plus lomustine versus lomustine alone in patients with recurrent anaplastic astrocytoma. At the time the study began in 2016, tumors from all enrolled patients were classified as grade 3 astrocytoma, but the 2021 WHO updates to tumor classifications later showed that the patients had different tumor types, including grade 3 and grade 4 IDH-mutant astrocytoma, and IDH-wildtype glioblastoma. When researchers analyzed results from the entire cohort, the combination treatment did not improve overall survival compared with lomustine alone. However, when they did a subset analysis specifically on patients with recurrent grade 3 astrocytoma that carried an IDH mutation, the results were significant. In this group, people who received the combination had a median survival of nearly 35 months, compared to 24 months with lomustine alone. Progression-free survival with the combination was almost 16 months, compared to roughly 7 months with lomustine alone. These meaningful survival gains have the potential to guide future clinical practice for treatment of recurrent grade 3 IDH-mutant astrocytoma. 

Join us next week on Monday, January 12th at 7pm EST for a webinar with Dr. Vijay Agarwal on "Sonodynamic Therapy: a Phase 2b Trial for Newly Diagnosed Glioblastoma." To join, visit virtualtrials.org/webinar.  

We are having a fundraiser event at Drive Shack in West Palm Beach, Florida on December 29th!  
Click HERE for details!

This was a very small early study done in India. They explored whether a combination of the supplements resveratrol and copper might affect glioblastoma biology. Ten patients received the supplements orally four times a day for roughly 11 days prior to resection surgery, and their tumor tissue was compared to samples from ten patients who did not. The treated tumors showed differences in several biomarkers linked to tumor aggressiveness, suggesting a possible biological effect on the tumor environment.

While these findings are interesting, there are important caveats. This was a small, short-term study that looked only at tumor tissue changes, not whether patients lived longer or felt better. Further research is needed to determine whether prolonged treatment with this combination can improve patient outcomes.

GE Healthcare, one of the largest MRI manufacturers in the US, is partnering with Imaging Biometrics (IB) to distribute advanced imaging software that can help doctors better distinguish brain tumor progression from treatment effects with "Fractional Tumor Burden" maps. This is encouraging news for the brain cancer community. We hope to see more widespread adoption of these tools that make it easier to monitor brain tumors and understand how they are responding to treatments. For a great webinar on Fractional Tumor Burden maps, click here. 

This new study from Japan provides further evidence in favor of adaptive radiation therapy (ART) for glioblastoma (GBM). The ART approach adjusts the radiation plan during treatment based on changes in the tumor or surgical cavity. In 59 GBM patients, ART was safe and showed promising outcomes: median overall survival was about 27 months, progression-free survival about 10 months, and serious radiation side effects were rare. While research on ART in brain cancer is still developing, it does provide hope that smarter, adaptable radiation plans could help patients live longer with fewer side effects.

Imvax reported top-line results from its randomized double-blind, placebo-controlled Phase 2b trial of IGV-001 in 99 patients with newly diagnosed (ndGBM). IGV-001 demonstrated a median overall survival (mOS) of 20.3 months, a 6.3 month improvement over placebo (14.0 months). Median follow-up was 22 months. The trial did not meet statistical significance for the endpoint of progression-free survival, but showed a very favorable profile with no drug-related serious adverse events. 

The study used 2:1 randomization across 19 U.S. sites, one of which was initiated by our Chief Scientific Advisor, Dr. Steven Brem! About 48 hours post-tumor resection, patients received biodiffusion chambers containing either personalized whole tumor-derived cell with an antisense oligonucleotide (IMV-001) or inactive solution (placebo); chambers were removed 48 hours later. All patients then received standard-of-care chemoradiation and maintenance temozolomide.

IGV-001 has Fast Track and Orphan Drug designations, and Imvax plans to meet with the FDA in the coming months to discuss the regulatory pathway. Given these significant results and the profound lack of promising options for GBM, we hope to see widescale patient access to this therapy in the near future. 

As we come to the end of the year, we're proud to share our Musella Foundation 2025 Highlights. Everything we do — funding research, expanding patient navigation services, providing vital educational resources, patient advocacy — can only be done with your support. Please consider making a year-end donation to help us continue driving progress, supporting patients and families, and bringing hope to the brain tumor community. Every dollar helps! 

A promising new combination approach from ImmunityBio, Inc. is gaining attention after one patient helped reveal its potential for recurrent glioblastoma (GBM). An initial study tested natural killer (NK)-cell immunotherapy combined with Anktiva, an engineered IL-15 agonist, plus bevacizumab; the trial allowed participants to use Optune but didn’t require it. However, when the first patient wearing Optune enrolled and did well on treatment, his unusually strong response suggested that the device may enhance how NK cells and Anktiva work together against GBM. These results prompted the launch of a further Phase 2b portion of the trial to see if the benefit can be replicated. This story highlights the important point that GBM immunotherapy trials should not exclude patients who want to use Optune.

Researchers at the University of Pennsylvania have found that a 70-year-old blood pressure drug, hydralazine, blocks an oxygen-sensing enzyme called ADO—a molecular “alarm” that controls blood vessel constriction. This discovery is relevant for brain cancer, because glioblastoma relies on the ADO pathway to survive in low-oxygen environments. When researchers tested hydralazine on glioblastoma cells in the lab, it pushed them into a non-dividing, dormant state known as senescence. While this finding is exciting, hydralazine has poor blood-brain barrier penetrance, so the next step will likely be development of new ADO inhibitors that are more tissue specific and better at crossing the blood-brain barrier.

New research provides the strongest evidence yet that bacteria are present inside both gliomas and brain metastases, expanding a trend seen in several recent studies. Two major efforts—from MD Anderson and the Weizmann Institute—used advanced imaging and genetic analyses to detect bacterial genetic material and molecules inside brain tumor cells, nearby immune cells, and surrounding tissue. Some of these signals resembled microbes normally found in the mouth or gut, suggesting they may travel through the body to the brain, though most appear to be fragments rather than fully living colonies. These new findings aren't entirely surprising, given that viral microbes, e.g. cytomegalovirus (CMV), have been documented in brain tumors for years.

These new studies show that brain metastases carry more diverse bacterial populations than glioblastomas, and location matters: posterior metastases show higher diversity than anterior ones. In glioblastoma, bacterial signals are less abundant but linked to metabolic pathways that may support tumor survival, while in metastases they’re associated with pathways tied to spread and invasion.

These microbial signatures correlate with immune and metabolic activity in the tumor, suggesting they may influence tumor behavior, immune responses, or therapy resistance. Some bacterial species even made tumor cells more resistant to chemotherapy in lab tests. Patients with higher bacterial loads in brain metastases tended to have shorter survival.

While the data so far does not prove causation between bacterial elements and tumor behavior, the presence of bacterial genetic material in brain tumors poses some interesting questions about systemic influence of the microbiome and could reshape how scientists view the tumor microenvironment. 

A longer-term analysis of the Phase 3 INDIGO trial was just published in The Lancet Oncology, and it confirms the durable benefit of Voranigo (vorasidenib)—the first targeted therapy FDA approved for Grade 2 IDH-mutant glioma in 2024. With an additional six months of trial follow-up, patients on Voranigo continued to show prolonged progression-free survival and delayed need for further intervention compared to placebo, along with sustained reductions in tumor growth and seizure frequency and no new safety issues. These data strengthen confidence that Voranigo offers meaningful and durable disease control for patients with low-grade IDH-mutant glioma. Use of the drug in Grade 3 or 4 IDH-mutant brain tumors remains investigational, but there are some ongoing studies that will hopefully yield evidence on the impact in higher grade tumors in the near future. 

Big news! The Musella Foundation teamed up with Kids Beating Cancer, the AYJ Fund, Tough2gether Foundation, DDRFA, and Springhood Ventures to help FLAG Therapeutics raise over $500,000 to push their promising drug, FLAG-003, toward clinical trials for kids with DIPG.

FLAG-003 has shown great results in preclinical testing. It crosses the blood-brain barrier and kills tumor cells through two powerful mechanisms, while sparing healthy cells. This could be a game-changer for DIPG and other brain tumors.

We're proud to be part of this initial funding, but we need to help them raise a lot more money to get this treatment to the kids who need it! If your organization wants to join in, contact me at musella@virtualtrials.org. Individuals can donate at virtualtrials.org/donate and mention it's for the FLAG project!

This new paper gives a nice in-depth review of sonodynamic therapy (SDT) using 5-ALA, the same compound many surgeons already use for fluorescence-guided surgery. In SDT, the 5-ALA builds up in tumor cells and, when activated by low-intensity focused ultrasound, triggers the release of reactive oxygen species that kill cancer cells while sparing healthy tissue. 

The review summarizes the strong preclinical data behind SDT therapy and details all of the human trials that have started so far, some of which have faced funding challenges. Because 5-ALA already has an excellent safety record and because many large medical centers already have focused ultrasound machines, this treatment has great potential for clinical adoption. That said, the approach is still experimental and the human studies so far are still working out important technical details, such as ultrasound dosing and timing. 

Multiple sites across several states are now enrolling patients for a Phase II trial testing a personalized dendritic cell therapy for glioblastoma (GBM). This therapy, called DOC1021 or Dubodencel, uses a sample of the patient’s own tumor to teach their dendritic cells what the cancer looks like, so the immune system can recognize and attack it. The therapy is described as "double-loaded" because it utilizes both tumor lysate and amplified tumor mRNA. In the earlier Phase I trial of 16 newly diagnosed GBM patients (15 of whom were unmethylated), the treatment showed a favorable safety profile and a 12 month survival rate of 88%. Notably, four patients remained alive at 22 to 33 months of follow-up. 

GT Medical Technologies announced interim results from its Phase 3 trial investigating the use of GammaTiles in patients with newly diagnosed operable brain metastases. GammaTiles are small, bioabsorbable collagen tiles with embedded radiation seeds that can be placed along the walls of the surgical cavity after tumor removal. They immediately begin delivering a low, targeted dose of radiation to the surrounding tissue. 

So far, the trial has shown that GammaTiles reduced the risk of tumor recurrence or death by 50% compared to standard of care (surgery followed by stereotactic radiosurgery [SRS] after recovery). Importantly, the rate of side effects was low and comparable in both groups. The interim results are based on 168 of the 230 patients enrolled in the trial, but we hope to see similar results as complete data from all enrolled participants matures. GammaTile is already FDA cleared for use in newly diagnosed and recurrent malignant brain tumors.

PinPoint Patient Recruiting, a market research recruitment company, is searching for people who have been diagnosed with glioblastoma (GBM), or their care partner, to participate in an online survey about their experiences.

If you or your loved one is over the age of 22, a resident of the US and is pursuing treatment, you may be eligible to participate. Those who qualify to participate in the study will receive $75 as a thank you. All information and responses will remain confidential. 

Interested?
To see if you qualify for the study or to get more information, please visit www.pinpointpatientrecruiting.com/gbm-survey-al or contact Kim Slusher at kim@pinpointpatientrecruiting.com.

DocDelta, a healthcare market research company, is seeking insights from patients and caregivers of individuals diagnosed with brain or spine cancer including a subset with a confirmed BRAF mutation. They aim to collect data capturing the real lived experiences of patients and caregivers through interviews and digital diaries in order to inform treatment development and patient support programs.

Who qualifies? Patients and caregivers of patients with BRAF mutation

Participant Compensation:
• 1 hour interviews ($120 per hour)
• 75 minute online diary during the week prior to interview ($150 incentive)
Total compensation for patients/caregivers is $270

How it works: Interested patients and caregivers will be sent a link with a screening criteria form. If the DocDelta team determines you are eligible to participate, they will schedule an interview and provide instructions for an online diary exercise to begin one week prior to the interview. To see if you qualify or get more information, please email DocDelta at patients@tdg.health or complete the screening criteria form HERE.