We had a technical issue sending out the news blast last week, so we apologize in advance if you receive a duplicate issue today.

We added a new webinar to our May lineup! In partnership with Mt. Sinai, please join us for "Empowering Brain Tumor Patients: Taking Control Through Knowledge, Support, and the Latest in Treatment Options" this Wednesday, May 21 at 5-6pm ET. This webinar will not be in our Zoom room; you can register here Mt Sinai webinar room. 

Our final May webinar will be Thursday, May 29 at 6pm ET, "Advancement of B7-H3 targeting CAR T cells against pediatric brain and spinal cord tumors" with Dr. Nicholas Vitanza 

Looking ahead, we also have an exciting webinar scheduled for Wednesday, June 25, 2025 (8pm ET) "Integrative Medicine for Patients with Primary Brain Tumors" with Dr. Nicholas Butowski 

A new study suggests that postoperative gabapentin, a neuromodulator usually taken for nerve pain, may improve overall survival in patients with newly diagnosed glioblastoma. Across two cohorts totaling 1,072 patients, those who received gabapentin lived a median of 4 months longer at Mass General Brigham and 6 months longer at UCSF compared to those who did not. Gabapentin targets TSP-1, a synaptogenic protein implicated in tumor progression. Importantly, the study findings showed that gabapentin use was associated with reduced serum TSP-1 levels. The study has limitations, including a retrospective design and variability in dosing across patients, but hopefully future prospective trials confirm these results and explore optimal dosing.

Congratulations and thank you to everyone who participated in the annual Minnesota Brain Tumor 5k over the weekend. The event was a big success, and all of the funds raised will be used exclusively for brain tumor research. Special thank you to event co-chair Melissa Dickhausen who helped share the event on the news!

Previously, craniectomy (removing a small portion of the skull) had been shown to increase penetration of tumor-treating fields (TTF) into the brain. However, the factors that modulate this effect were still unknown. This recent article shows that the biomaterials used to fill the opening, the gross tumor volume relative to the opening size, and the amount of edema around the tumor are important factors. These insights may help optimize the application of TTFields for better treatment results in the future.  

The 16th Annual Minnesota Brain Tumor 5K is this weekend! Event co-chair, Melissa Dickhausen, did a fantastic job sharing the event and raising awareness about brain tumors on the news. For more information about the event or to sign up, click here! 

Our May webinar series continues! Please note that this Thursday's will be at 7pm EDT (it was previously scheduled for 6pm). To join, go to virtualtrials.org/webinar!

Thursday, May 15 @ 7pm EDT: "Fractional Tumor Burden Mapping" with Dr. Leland Hu

Sunday, May 18 @7pm EDT: "Glioblastoma: Beyond the Guidelines" with Dr. Burt Nabors

Thursday, May 29 @6pm EDT: "Advancement of B7-H3 targeting CAR T cells against pediatric brain and spinal cord tumors" with Dr. Nicholas Vitanza
 

Mark your calendars! To join, visit virtualtrials.org/webinar.

Tuesday, May 6 @ 7pm EDT: "Finding Clinical Trials" with Unsha Bakker, MSN, RN, OCN, CBCN, Brain Cancer Support and Solutions Alliance Nurse Navigator

Wednesday, May 7 @ 7pm EDT: "New strategies to bypass the blood brain barrier to improve the delivery of chemotherapeutics for brain tumors" with Dr. John Boockvar 

Thursday, May 15 @ 6pm EDT: "Fractional Tumor Burden Mapping" with Dr. Leland Hu

Sunday, May 18 @ 7pm EDT: "Glioblastoma: Beyond the Guidelines" with Dr. Burt Nabors

Thursday, May 29 @ 6pm EDT: "Advancement of B7-H3 targeting CAR T cells against pediatric brain and spinal cord tumors" with Dr. Nicholas Vitanza

The FDA recently granted breakthrough therapy designation to a B7-H3-targeting CAR-T therapy for the treatment of diffuse intrinsic pontine glioma (DIPG). The CAR-T therapy is delivered directly into the brain via repeated intra-cerebroventricular doses. The FDA decision was based on encouraging data from the Phase 1 BrainChild-03 study conducted by Seattle Children’s, showing a median survival of 10.7 months from first CAR-T dose and 19.8 months from diagnosis among 21 DIPG patients, with three patients still alive at 44, 45, and 52 months post-diagnosis. There are now plans to launch a pivotal phase 2 trial later this year. We'll be having a webinar with Dr. Nicholas Vitanza about this therapy on May 29!

Researchers at Mass General Brigham, in collaboration with Boston Children’s Hospital and Dana-Farber, developed an AI model using a method called temporal learning to predict tumor recurrence in children with gliomas. The model analyzes sequences of post-treatment scans to detect subtle changes over time, achieving prediction accuracy rates of 75% to 89%. The model's accuracy plateaued between 4 to 6 scans. It's worth noting that the study was made possible by access to nearly 4,000 scans from 715 pediatric patients, with imaging and clinical data access support from the Children’s Brain Tumor Network (CBTN).

We have some great webinars coming up in the next few weeks for May Brain Cancer Awareness Month!

Tuesday, May 6 (7pm ET) "Finding Clinical Trials" with Unsha Bakker, MSN, RN, OCN, CBCN, Brain Cancer Support and Solutions Alliance (BCSSA) Nurse Navigator

Wednesday, May 7 (7pm ET) "New strategies to bypass the blood brain barrier to improve the delivery of chemotherapeutics for brain tumors" with Dr. John Boockvar

Thursday, May 15 (6pm ET) "Fractional Tumor Burden Mapping" with Dr. Leland Hu

Sunday, May 18 (7pm ET) "Glioblastoma: Beyond the Guidelines" with Dr. Burt Nabors

You can join any or all of these webinars by visiting virtualtrials.org/webinar. No pre-registration is required!

In the Phase 2 IPAX-LINZ study conducted in Austria, eight patients with recurrent high-grade glioma (first or second recurrence) received intravenous TLX101 before and after second-line external beam radiation therapy (EBRT). TLX101 targets the LAT1 amino acid transporter, which is commonly overexpressed in GBM. The treatment was well tolerated, with no serious adverse events, even at higher doses than previously tested. The median overall survival was 12.4 months from treatment start, which is an improvement over the typical 9.9 months for recurrent GBM treated with EBRT alone. 

Telix has an ongoing Phase 2 trial for TLX101 in newly diagnosed GBM (NCT05450744) in Australia, New Zealand, Austria and the Netherlands, and plans are underway for a pivotal trial in recurrent GBM which will hopefully open enrollment in Australia in late 2025 (followed by US site activation and enrollment pending FDA IND approval). 

This new study from St. Jude Children’s Research Hospital shows that the FOXR2 gene—once thought to be specific to CNS neuroblastoma—is actually active in several other types of pediatric brain tumors too, including aggressive forms like high-grade gliomas and pineoblastomas. In a review of 42 brain tumors with FOXR2 activation, only a quarter were true CNS neuroblastomas. Despite similar levels of FOXR2 activity across tumor types, outcomes varied drastically: children with CNS neuroblastoma responded well to treatment, but those with gliomas or pineoblastomas had very poor survival outcomes.

This study shows that physicians should not use FOXR2 activation as an exclusive marker for CNS neuroblastoma and instead should use comprehensive molecular testing, histology, and imaging to make accurate diagnoses and treatment plans.  

The drug Jobevne is now FDA approved for intravenous use in multiple cancer types, including glioblastoma. We cover this drug in our copay assistance program, as a bioequivalent to Avastin.

VMX01 is an oral bacterial vaccine designed to stimulate VEGFR2-specific killer T-cells, which disrupt the tumor's blood supply and enhance immune cell infiltration. In this Phase 2a study, the combination of VMX01 and avelumab (a PD-L1 inhibitor) for recurrent glioblastoma (GBM) was generally well tolerated. In the total evaluable study population (n=25), median progression free survival was 2.7 months and median overall survival was 11.1 months. Among patients with resected recurrent disease, overall survival ranged from 2.2 months to 46.5 months. Notably, the study investigators identified potential pharmacodynamic and predictive biomarkers related to tumor response that could be leveraged in future trials. 

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 Phase 1 trial for oral gallium maltolate (GaM) in recurrent GBM is not yet complete but preliminary results were shared in a press release today. Oral GaM is taken in pill form daily and has demonstrated excellent tolerability, with no serious adverse events reported. Among the 23 patients in the study so far with evaluable data, overall survival was 14 months from GaM treatment initiation and 32 months from the time of initial diagnosis. Median overall survival for recurrent GBM is typically 8-9 months, so this represents a positive signal of response. It’s worth noting that this was a dose escalation study, so not all patients received the maximum dose.

The data also showed progression-free-survival (PFS) of 2 months, which is comparable to patients undergoing standard therapy for recurrence. PFS in the study was determined by standard radiologic (RANO) response criteria, which doesn’t always reliably distinguish tumor progression from treatment effect. The company that makes oral GaM has developed advanced imaging software called Fractional Tumor Burden mapping (FTB maps) that will hopefully provide more insight for imaging interpretation in the future. We have a webinar on FTB maps with Dr. Leland Hu coming up on May 15!

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!

This is interesting preclinical work from researchers at UT Health San Antonio. Radiation therapy, while effective at shrinking glioblastoma tumors, can unfortunately help drive recurrence by inducing senescence in cancer cells. These senescent cells secrete growth factors that later promote the regrowth of surviving tumor cells. However, the UT researchers found that administering the senolytic drug birinapant after radiation selectively eliminates these senescent cells by targeting the anti-apoptotic protein cIAP2. In their mouse models, this approach (radiation followed by birinapant) delayed or prevented recurrence without harming healthy cells; notably, 2 out of 9 mice (22%) survived beyond 120 days with no tumor recurrence. This new study helps build on earlier research on senolytics in glioblastoma, but birinapant’s specificity for cIAP2 is a novel advancement. We hope to see continued research on this approach.

This preclinical study helps explain why some cancer cells may be less responsive to Tumor Treating Fields (TTFields). The researchers found that TTFields can activate the PI3K/AKT pathway over time, partly through proteins like FAK and N-cadherin. Importantly, in both cell assays and animal models, blocking the PI3K pathway with inhibitors made cancer cells more sensitive to TTFields.