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. 

Our next webinar is coming up on Monday, November 10, 2025 at 1pm ET. The topic is "Molecular Work-Up and Therapeutic Targeting of H3K27M-Diffuse Midline Glioma" with Dr. Carl Koschmann. To join, visit virtualtrials.org/webinar.  

A new study out of Roswell Park has provided insight on why some glioblastoma (GBM) patients have responded especially well to SurVaxM, an experimental vaccine that targets survivin. Survivin is a protein found in about 95% of GBMs, and the vaccine teaches the immune system to attack survivin-positive cells.

In the Phase 2a trial, 63 newly diagnosed patients received standard care plus SurVaxM. Researchers analyzed detailed molecular tumor data from 34 of those patients and found that long-term survivors (those living more than 18 months) had tumors with high B and T cell infiltration, stronger interferon signaling, and a shared five-gene expression pattern. These immune features didn’t predict survival in an external control group of registry patients who didn’t get SurVaxM, which suggests these molecular signatures might help predict who benefits most from the vaccine rather than just who has a better prognosis in general. 

Notably, ten patients are still alive over seven years after receiving SurVaxM, including five with no recurrence to date. It is also worth noting that both the Phase 2a and 2b trials only enrolled patients with very little tumor left after surgery (less than 1.0 cm3 contrast-enhancing disease), so these findings may not apply to everyone with GBM.

The larger Phase 2b randomized trial is now fully enrolled. An independent interim review from early 2025 recommended the trial continue without changes, which is an encouraging sign. We’ll share updates as soon as final results are available!

Researchers at CoSyne Therapeutics have discovered that glioma cells with non-mutant (wild-type) TP53 AND RB1 genes may be sensitive to cytarabine, which is a chemotherapy drug already approved for intrathecal injection in certain types of leukemia involving the brain and spinal fluid.

This finding suggests that cytarabine could be worth further study in patients with leptomeningeal spread of glioma, where chemotherapy is sometimes administered directly into the spinal fluid. CoSyne is interested in collecting case reports from patients who have both malignant glioma and leptomeningeal disease to better understand responses and tolerability in this rare condition. 

If you have this condition, it might be worth checking your pathology report for TP53 and RB1 genes. If they are wildtype (not mutated), this may be an accessible option for you. If you or your medical care team would like to learn more about this research effort, please sign up for our patient navigation program here. 

Our next webinar will take place on Monday, November 10, 2025 at 1pm ET. The topic is "Molecular Work-Up and Therapeutic Targeting of H3K27M-Diffuse Midline Glioma" with Dr. Carl Koschmann. To join, visit virtualtrials.org/webinar. 

NanO₂ is an investigational therapy being developed by NuvOx for newly diagnosed glioblastoma (GBM). It helps deliver oxygen into low-oxygen (hypoxic) tumor regions, making radiation more effective. It's given intravenously (just before each radiation session). The ongoing Phase IIb RESTORE trial for this therapy completed enrollment of 87 patients last month, and the company was recently awarded NIH/NCI funding to support advanced liquid biopsy work within the Phase IIb trial. We're glad to see this therapy advancing and hope to see positive results from the RESTORE trial within the next year.

 Final results from Novocure's Phase 3 METIS trial show that tumor treating fields (TTFields), when used after stereotactic radiosurgery (SRS) for brain metastases from non-small cell lung cancer (NSCLC), significantly delays intracranial progression compared to best supportive care alone. Patients receiving TTFields plus best supportive care had a median time to intracranial progression of 15 months, versus 7.5 months for the control group, with no negative impact on quality of life or neurocognition. For patients who received immune checkpoint inhibitors in addition to TTFields, the benefit was even more pronounced. While overall survival and neurocognitive outcomes were similar between groups, TTFields was well tolerated, with primarily low-grade skin-related side effects. Based on these results, Novocure plans to apply for FDA approval of TTFields for brain metastases from NSCLC. 

Researchers in Japan have developed a new, ultra-fast system (GeneSoc®-based rapid phenotyping system) that can detect key genetic mutations in brain tumors in under 25 minutes. Their research, recently published in Neuro-Oncology, showed that the system exhibits high diagnostic accuracy, with 98.5% sensitivity and 98.2% specificity for detection of ID1 mutations, as well as 100% sensitivity and specificity for detection of TERT promoter mutations. This system may not only be effective for assisting with rapid diagnosis, but also for detecting tumor boundaries intraoperatively. While clinical adoption of this type of technology may take time, this is a promising step forward. 

A new paper published in Neuro-Oncology Advances introduces the first-ever Guiding Principles for Adult CNS Tumor Treatment Programs in the United States. This was a joint effort among several brain tumor patient advocacy organizations, including the Musella Foundation. These principles fill a critical gap by defining what specialized, comprehensive care for brain and CNS tumor patients should include, such as expert multidisciplinary teams, molecular testing, access to clinical trials, and supportive services. The full guidelines are now publicly available to help patients and caregivers make more informed treatment decisions.

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"!