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. 

Dr. George Lundberg, Cancer Commons Editor in Chief, and Dr. DeLeys Brandman, Cancer Commons Medical Director, recently shared a discussion on the 'Learning Health System' model utilized by Cancer Commons, plus a new platform technology - CRO-LITE - that will help support fast, high-quality n-of-1 clinical trials. The Musella Foundation and Head for the Cure have partnered together with Cancer Commons to provide this service to brain tumor patients. For brain tumor patients and caregivers seeking navigation services, click here. 

Avapritinib is FDA approved for stromal tumors and systemic mastocytosis. A collaboration across three institutions has now shown this drug may have promise in high grade glioma (HGG) because it can cross the blood-brain barrier and target PDGFRA mutations, which occur in about 15% of pediatric HGG cases. Based on positive preclinical results, the drug was given to a small group of patients through an expanded access program, and three out of seven had tumor shrinkage. Thanks to these positive real world results, HGG was included as an eligible condition for an ongoing Phase 1 pediatric trial testing the drug in multiple solid tumor types. 

A new study has reinforced previous research linking firefighting with cancer development. The study, published in Cancer, showed that glioma tumors in some firefighters exhibited a unique genetic signature associated with exposure to haloalkanes, chemicals found in flame retardants and fire extinguishants. The research compared tumors from firefighters and non-firefighters, showing more frequent haloalkane-related mutations in the firefighters' tumors. While the study is small, it suggests environmental chemicals may play a role in glioma development.

Interim analysis from the Phase 2/3 CLINGLIO trial suggests that adding the investigational therapy LAM561 to standard treatment may improve progression free survival (PFS) in newly diagnosed glioblastoma patients with MGMT methylation. While LAM561 was generally well tolerated, it didn't significantly improve PFS in the overall study population. However, in GBM patients with MGMT-methylated tumors, the LAM561 group had a median PFS of 86.4 weeks compared to 54.7 weeks for standard of care alone. Final trial results, including the drug's effect on overall survival, are expected by the end of 2026.

Hoops for Hannah is back for another year! This contest raises funds for brain tumor research in memory of Hannah Taylor. Contest entry is free, but you can also donate to our organization. There is a $1,000 prize! For details to enter, click here. 

This is one of the more promising investigative treatments currently in trials for recurrent GBM. We did a webinar with Dr. Marc Hedrick on this treatment last year, where he shared some of the Phase 1 results and preliminary data from the Phase 2 trial. The full Phase 1 results are now published and available here. Information on the Phase 2 trial, which is currently recruiting, can be found here. 

We have a webinar tomorrow, March 11, at 1pm EST. Dr. Soma Sengupta will discuss the Gallium Maltolate Expanded Access Program for glioblastoma. To join, visit virtualtrials.org/webinar.  

For those following the story of Professor Richard Scolyer, world-renowned pathologist and melanoma researcher who was diagnosed with GBM in 2023, this case report related to his treatment was just published. The article mentions that a clinical trial testing neoadjuvant combination checkpoint inhibitors (ICIs) is planned for newly diagnosed GBM. For more info on the planned clinical trial, click here.

This is interesting new research from UCLA. They found that a plant-derived compound called forskolin can cross the blood-brain barrier and can be used synergistically with radiation. Recent research has shown that radiation may temporarily make glioma stem cells more flexible, so researchers theorized that adding forskolin at the right moment could exploit this flexibility by pushing the cells towards a less harmful (i.e. nondividing) neuron-like or microglia-like state. When they tested the dual combination in mice, the approach extended median survival and, in some cases, led to long-term tumor control.

The Musella Foundation helped fund this research! This recently published preclinical study shows how changing the conductivity of the hydrogel used with tumor treating fields (TTFields) can improve the therapy effectiveness by increasing coverage of gross tumor volume up to a certain point. This research will help optimize this aspect of TTFields while also minimizing skin toxicity. The article also gives some good insights on potentially improving scalp/skin conductivity.