Mark your calendars for Tuesday, March 3rd at 7pm ET for a webinar with Dr. Robert Aiken on: "Clinical trial of a new individualized dendritic cell vaccine for treatment of newly diagnosed glioblastoma." 

In a new study published in Science Translational Medicine, researchers led by Dr. Hui Li at the University of Virginia School of Medicine report a first-in-class small-molecule inhibitor targeting AVIL, an oncogene previously shown to drive glioblastoma growth. Discovered through high-throughput screening, the molecule crosses the blood–brain barrier and suppressed tumor growth in multiple cell and mouse models. AVIL is mostly absent in normal brain tissue, and the molecule did not significantly affect healthy brain cells in preclinical studies, supporting its potential selectivity. While the current molecule form is not yet a finished drug, we're eager to see these researchers finalize chemical optimization of the compound and move into human trials within the next year or two.

Novocure's Tumor Treating Fields (TTF) technology first earned FDA approval for recurrent glioblastoma in 2011 and for newly diagnosed glioblastoma in 2015 (Optune Gio). While some in the neuro-oncology community have been skeptical of TTF, the technology went on to gain FDA approval for malignant pleural mesothelioma in 2019 and for metastatic non-small cell lung cancer in 2024 (Optune Lua).

This past week, Novocure received FDA approval for TTF for locally advanced pancreatic cancer, used in combination with gemcitabine and nab-paclitaxel (Optune Pax).

While TTF by itself is not a cure for brain cancer, the steadily expanding list of FDA-approved indications reinforces that this technology has demonstrated meaningful clinical efficacy across multiple solid tumor types. 

The GIANT trial has begun enrolling in the United States to test whether neoadjuvant immunotherapy can improve outcomes for people with newly diagnosed glioblastoma (GBM). Led by Dr. Mustafa Khasraw at Duke University, GIANT is a Phase 2 trial evaluating the immune checkpoint inhibitors nivolumab and relatlimab given before surgery, followed by these agents with standard radiotherapy and temozolomide afterward.

Unlike most prior GBM immunotherapy studies, this trial focuses on giving immunotherapy early (before and after initial surgery) based on the idea that an intact, treatment-naive tumor might be more responsive to immune stimulation. Interest in the trial has been heightened by high-profile patient advocates and researchers, including world-renowned melanoma expert Professor Richard Scolyer, who has lived well beyond typical expectations after his own GBM (MGMT unmethylated) diagnosis. More info on the trial can be found HERE.

Good news! The National Comprehensive Cancer Network (NCCN) patient guidelines are free, evidence-based resources written in plain language that reflect expert consensus cancer treatment guidelines used by clinicians worldwide to support decision-making. NCCN usually updates the patient guidelines for each cancer category every few years, but they just recently announced that all patient guidelines will be updated annually to better keep pace with advances in cancer care and expand access across multiple languages. This is a positive step forward; the last NCCN patient guidelines for gliomas was issued in 2024 (see here).

Researchers from the University of Michigan have reported a new metabolic vulnerability in pediatric ependymoma, a common childhood brain tumor that remains incurable with current therapies. Standard treatment today is maximal surgical resection followed by radiation, with chemotherapy offering limited benefit and no approved targeted therapies. In this new study, the investigators showed that most supratentorial ependymomas driven by the ZFTA-RELA fusion protein depend on production of itaconate, a metabolite usually made by immune cells. They found that tumor cells generate itaconate via the enzyme ACOD1, and that itaconate and ZFTA-RELA form a feed-forward loop that promotes tumor growth through glutamine metabolism. Blocking this pathway reduced fusion protein levels and shrank tumors in mouse models, providing the first evidence that ZFTA-RELA-driven ependymomas can be targeted indirectly. The researchers are now working with the Pediatric Neuro-Oncology Consortium (PNOC) to develop a clinical trial targeting the itaconate pathway.

A promising new pediatric brain tumor trial that will test personalized mRNA cancer vaccines is opening across multiple sites in Australia. The PaedNEO-VAX trial, funded by Providence Therapeutics alongside the Australian government and philanthropic partners, will enroll children and adolescents with recurrent or progressive high-grade glioma, diffuse midline glioma, medulloblastoma, and ependymoma.

Using tumor genome sequencing, each child’s cancer will be analyzed to identify individualized targets, followed by manufacture of a custom mRNA vaccine using Providence’s platform, with an anticipated turnaround of about 10 weeks from enrollment to dosing. Phase I will assess safety and dosing, and Phase II will evaluate progression, survival, and quality of life outcomes. The trial builds on Providence’s prior compassionate-use experience, including treatment of the company founder’s son with a personalized mRNA vaccine.

Providence has expressed interest in working with partners in the United States and Canada, including patient advocacy organizations, to explore launching a similar North American trial.

If you are a brain or spinal cord tumor survivor, living in the NY/NJ/CT area, you are eligible to apply for a Making Headway college scholarship. In 2026, Making Headway will offer at least 15 scholarships, each worth up to $5,000. A special $15,000 scholarship will also be given as part of the 7th annual Michael Schwartz Making Headway Scholarship.

To be eligible for consideration, each applicant must:
1. Be a brain or spinal cord tumor survivor (diagnosed before the age of 18)
2. Reside in the New York City metropolitan area (including New Jersey and Connecticut)
3. Have been admitted to, or be currently attending, a two- or four-year college or vocational program.

For additional requirements, to download the application, or apply online, visit http://makingheadway.org/scholarship.

The deadline for the applications to be submitted in May 1, 2026. If you have any questions, please email Jennifer@MakingHeadway.org.

Kids and adults living with brain tumors - we invite you to submit original painting/drawings through your unique lens. Selected pieces may be featured in our upcoming 13th edition of the Musella Foundations Brain Tumor Guide to help raise awareness and inspire others facing similar paths.

Please submit your high resolution image that will appear in black & white to: Musella@virtualtrials.org. 

We're excited to share that the Musella Foundation Copayment Assistance Program is now OPEN to new patients again! After being temporarily closed to new applicants, we are once again accepting applications and helping patients access needed treatments. 

What's new? 
- We've added the drug Modeyso to our list of covered treatments
- We've increased the income eligibility guidelines, allowing more families to qualify for assistance

Cancer is hard enough — worrying about treatment costs shouldn't be part of the burden. Our goal is to reduce financial barriers so patients can focus on their care. For more information and to apply, go to braintumorcopays.org.

Another encouraging update on GB13, a targeted immunotoxin that selectively kills brain tumor cells that express the IL13Ra2 receptor, which is found on many DIPG tumors but is largely absent from normal brain tissue.

In a newly published preclinical animal study, GB13 significantly reduced tumor size and improved survival across multiple DIPG models. The results were especially compelling when GB13 was given before and alongside radiation therapy. Notably, durable (and in some cases curative) responses were observed in human tumor–derived DIPG models. Researchers found that GB13 interferes with tumor cells’ ability to repair radiation-induced DNA damage, making radiation more effective at killing cancer cells. This radiosensitizing effect was consistent across multiple patient-derived DIPG cell lines and validated in both immunocompromised and immunocompetent mouse models. Together, these findings strongly support Targepeutics’ planned IND submission and upcoming Phase I clinical trial, with an expansion Phase II component, which will evaluate GB13 in children with DIPG.

As we’ve shared previously, The Musella Foundation helped support the early research behind GB13 more than a decade ago. It’s exciting to see this work continue to move steadily toward the clinic!

We previously hosted a webinar on Carthera's SonoCloud trial, which uses a skull-implantable ultrasound device to open the blood-brain barrier so chemotherapy can better reach glioblastoma (GBM) tumors. Now, a new published study has shown that an investigational liquid biopsy tool (a blood test that detects cancer material circulating in the bloodstream) was able to detect tumor-derived vesicles released after paclitaxel chemotherapy treatment and that the detection of these vesicles correlated with survival. While more prospective validation is needed, these early findings give hope that doctors may soon have a real-time way to see whether this therapy is working.

The Musella Foundation is a proud supporter of Penn Medicine's 13th Annual Neuro-Oncology Brain Tumor Symposium taking place this Friday, January 30th. 

This event will cover the latest advances in state-of-the-art care for treatment of brain tumors. The symposium is designed for neurosurgeons, neurooncologists, radiation oncologists and all healthcare professionals involved in the treatment of patients with brain tumors. Patients, caregivers, patient advocates, and members of the public who may benefit from understanding current innovative approaches to brain tumor care are also invited. 

If you are interested in attending in-person or virtually, register HERE! 

The Alliance A071401 trial tested abemaciclib, an oral CDK4/6 inhibitor already approved for certain breast cancers, in patients with grade 2 or 3 recurrent or progressive meningiomas whose tumors carried NF2 or CDK pathway mutations. All patients had already been treated with surgery, radiation, or both, and had no standard treatment options remaining.

In this study, the first 24 treated patients received a median of nine cycles of abemaciclib. At six months, 58% of patients had no tumor progression, a good result when compared with prior studies showing only 0–29% of patients typically remained progression-free at that time point. Median progression-free survival was 10 months, and median overall survival was 29 months. These outcomes suggest meaningful disease stabilization in a population with very limited alternatives.

ImmunityBio’s combination approach for recurrent glioblastoma (GBM) continues to show early promise. The Phase 2 QUILT-3.078 trial is testing NK-cell immunotherapy with Anktiva (an engineered IL-15 agonist), bevacizumab, and Optune. So far, 19 of 23 patients treated on the trial are still alive, and some have gone 12 months or more from recurrence without disease progression. Patients started the trial with very low lymphocyte counts from prior treatment, but the therapy helped restore immune function, and side effects have been manageable. While these results are still very early, we are watching this trial closely and hope to see further updates over the next few months! 

Kids and adults living with brain tumors - we invite you to submit original painting/drawings through your unique lens. Selected pieces may be featured in our upcoming 13th edition of the Musella Foundation's Brain Tumor Guide to help raise awareness and inspire others facing similar paths.

Please submit your high resolution image that will appear in black & white to: Musella@virtualtrials.org.

Good news from IN8bio! They recently announced positive trends from their Phase 1/2 trial of gamma-delta T cell therapy in newly diagnosed glioblastoma. Patients treated with repeated doses had a median progression-free survival of 13 months, nearly double that seen with standard of care. Median overall survival has not been reached, but it exceeds 17 months so far. Several treated patients remain progression-free beyond two years, and the therapy has been well tolerated, with no serious treatment-related toxicities reported. 

GammaTile is a small implant placed in the brain at the time of resection surgery that delivers radiation right away instead of waiting weeks for standard post-surgery radiation. It was first cleared by the FDA in 2018 for both newly diagnosed and recurrent glioblastoma (GBM) and has been used in patients for several years. Now, GT Medical Technologies has started enrolling patients in the BRIDGES trial, a large randomized study to see whether GammaTile helps people with newly diagnosed GBM live longer compared with the current standard of care radiation. 

Researchers at the University of Michigan developed a machine-learning based “digital twin” program that maps how individual brain tumors use nutrients. Published in Cell Metabolism, the approach integrates patient blood data, tumor genetics, and limited metabolic measurements taken during surgery, including isotope tracing, to predict tumor-specific metabolic dependencies. Some tumors rely on particular amino acids or pathways and may be slowed by dietary changes or drugs, while others can bypass these restrictions.

The researchers validated the model using data from glioma patients who received labeled glucose during surgery and confirmed predictions in mouse models. The digital twin accurately identified which tumors would respond to the drug mycophenolate mofetil, while also distinguishing tumors that could evade the drug by using alternative “salvage” pathways to obtain DNA-building materials.

This is exciting work, and we hope to see further development and validation!

Researchers at Memorial Sloan Kettering found that PGBD5, a gene known to drive pediatric brain cancers by cutting and rearranging DNA, also plays an essential role in normal brain development. The study shows that PGBD5 deliberately creates DNA breaks in developing neurons that are precisely repaired to support proper neuron formation and function. This makes PGBD5 a rare “double-edged” gene whose beneficial developmental role can, when misregulated, lead to childhood cancers and neurodevelopmental disorders.

This important finding will hopefully lead to future development of therapies that can block PGBD5’s cancer-causing activity while preserving its normal role in brain development, potentially leading to safer treatments for pediatric brain tumors.