September is Childhood Cancer Awareness month, and pediatric brain tumors still remain some of the most difficult cancers to treat. Pediatric brain tumors are very different from adult brain tumors, they are genetically different and are located in different parts of the brain, often in areas that are too difficult to reach, making surgical resection challenging or impossible. Additionally, young, developing bodies and brains can be more vulnerable to many cancer treatments, resulting in life-long side effects. While a portion of the research on adult brain tumors may benefit children, much of that research will not target the unique traits found in pediatric brain tumors.
The American Brain Tumor Association recognized the need for research geared towards pediatric brain tumors decades ago. Since then, the ABTA has supported more than 100 pediatric brain tumor research projects, and many more projects that span both pediatric and adult tumors.
Two ABTA-funded projects have led to currently active immunotherapy clinical trials for pediatric brain tumors. ABTA also supported a study for a new therapy which recently received FDA Rare Pediatric Disease designation for two different types of pediatric brain tumors, with clinical trials currently in development.
The largely preclinical research that the ABTA funds is key to unlocking new treatments, but it takes time for that work to reach patients. These breakthrough studies took seven to fifteen years from the completion of the initial ABTA funding to reach these milestones. Furthermore, no single therapy will work for every patient. Therefore, the ABTA continues to look ahead and continues to fund this vital research.
A few of the more recently funded ABTA grantees, whose focus is on pediatric brain tumors, shared how meeting pediatric brain tumor patients early in their careers inspired them to pursue research in this field and find new treatments to help these young patients.
Mariella Filbin, MD, PhD – Dana-Farber Cancer Institute, Harvard University
Dr. Mariella Filbin’s research career began as a medical student when she met a young brain cancer patient. Meeting this patient began a 25-year career where her lab focuses on studying tumors like high-grade glioma and others that are in need of novel therapeutic options. Dr. Filbin’s ABTA research aims to understand the underlying biochemistry of Diffuse Midline Gliomas that allows the cancer cells to survive radiation treatment. She hopes to find new treatments to prevent radiation resistance, to improve outcomes for pediatric patients.
Robyn Gartrell, MD – Johns Hopkins University
Dr. Robyn Gartrell’s interest in pediatric brain tumors began in college after meeting a diffuse intrinsic pontine glioma (DIPG) patient at a dance-a-thon to raise money for pediatric cancer. Meeting that patient inspired her to pursue a career in biomedical research. She now leads a lab that studies combining radiation, chemotherapy, and immunotherapy to improve treatment responses and help prevent toxic side effects in pediatric cancer. Her ABTA research project uses a novel radiation strategy called FLASH, which minimizes injury to the normal, still developing, brain in young children with atypical teratoid/rhabdoid tumors (AT/RT).
John Prensner, MD, PhD – University of Michigan Medical School
Dr. John Prensner is a pediatric oncologist whose research lab studies the cancer genome in childhood brain tumors. He developed an interest in how cancers develop in young patients when he was a college student. While shadowing physicians, he met several pediatric patients. He was surprised that children so young could develop such aggressive brain cancers and he wanted to understand why this happens. His research now focuses on the genes in brain cancers like medulloblastoma, AT/RT, and DIPG and how these cancers begin. His ABTA grant studies the “dark proteome” looking at unique proteins that are created by the cancer cells and how they drive cancer progression. He hopes to find novel therapies targeting these novel proteins to help treat these aggressive cancers.
The ABTA remains committed to investing in the most promising pediatric research projects. We are proud to back dedicated scientists like Dr. Filbin, Dr. Gartrell, and Dr. Prensner, who work every day to rewrite the future of pediatric brain tumors. Together with our partners and community donors, we can fund the breakthroughs that give children a childhood free from cancer.
