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.
Our copayment assistance program is now closed to new patients and renewals. We will let you know when we reopen. Of course, we will continue to pay claims for those with active grants. We had a busy start to the year. In January alone, we approved 50 patients for grants up to $5,000 each, which is more than we had any month last year!
We're excited to see this special FDA designation for GB13! GB13 is an investigational immunotoxin which uses a precision-targeting mechanism that selectively attacks cells expressing IL13Ra2, a receptor that is highly expressed in brain tumors and other cancers but rarely found in normal tissues. The Musella Foundation gave 2 research grants to help get this therapy started and have been following it closely.
These two recently published studies represent a breakthrough in understanding and leveraging the role of OLIG2 in Sonic Hedgehog medulloblastoma (SHH-MB), and this work could potentially be applied to other brain cancers as well.
In the first study, Desai et al identified OLIG2 as a key regulator of the transcription from quiescent to activated states in SOX2+ stem-like cells, which is critical for tumor growth. They showed that inhibiting OLIG2 with CT-179 effectively blocks this transition, reducing tumor formation and regrowth after therapy.
The second study by Li et al. looked at CT-179's mechanism of action, showing it disrupts the ability of OLIG2 to pair up and bind to DNA. Their research revealed that CT-179 promotes tumor cell differentiation and apoptosis, enhances radiation, and could be combined with CDK4/6 inhibitors to overcome treatment resistance.
Based on these findings, we hope to see further development of CT-179 for SHH-MB and other brain tumors!
The Making Headway Foundation has a 2025 college scholarship application open! If you are a brain or spinal cord tumor survivor, living in the NY/NJ/CT area, you are eligible to apply. In 2025, at least 15 scholarships will be offered, each worth up to $5,000. A special $15,000 scholarship will also be given as part of the 6th annual Michael Schwartz Making Headway Scholarship.
The Making Headway Foundation college scholarship program is built upon the Scott J. Reisser Memorial Scholarship Fund, established in 2007. This fund is the culmination of Scott’s family’s efforts to create a fitting memorial to their beloved son, brother, grandson, nephew, and friend to so many. Scott’s most cherished dream was to graduate from college, a milestone he fell just short of achieving. After his death in 2006, it was clear that the most appropriate way to honor his memory would be by helping other young brain tumor survivors achieve their educational goals. Since 2007, Making Headway has given out 176 college scholarships in his memory.
To learn more and apply, visit http://makingheadway.org/scholarship/. The deadline for applications to be submitted is May 1, 2025. If you have any questions, please email Jennifer@MakingHeadway.org or call 914-238-8384.
Our first webinar of 2025 is tomorrow, January 28, at 7pm EST! Dr. Adam Sonabend will discuss his work with the Sonocloud-9 device and the Sonobird Phase 3 trial. To join, visit virtualtrials.org/webinar.
This presentation by Dr. Al-Gizawiy at the Medical College of Wisconsin includes some 'hot off the press' preclinical research on the potential for gallium maltolate as a radiosensitizer. There is also a good Q&A section at the end.
The Musella Foundation Copayment Assistance Program is running low on funding and will close to new and renewal applications soon. Of course, we will continue to pay claims for those already approved - just will not be able to accept new applications. So if you have been thinking of applying, go to brainumorcopays.org and follow the directions to send in an application!