Funded Grants

RENEWAL F.L.A.G. GRANT

Grantee: University of Southern California
Project Lead: Josh Neman, PHD
Grant Title: The role of GABA transaminase in medulloblastoma local recurrence and its potential as a therapeutic target in medulloblastoma metastasis
Program Area: Medulloblastoma - Pediatrics
Grant Type: F.L.A.G Grant
Year Awarded: 2022 and 2023
Amount: $100,000
Duration: 2 years

Summary: Medulloblastoma is a pediatric brain tumor that has a propensity to locally recur and to spread to other regions of the brain and spinal cord. Patients with these diagnoses will almost always succumb to their disease, highlighting the importance of performing research on these phenomena. When medulloblastoma tumors recur, cancer cells that were left over from tumor resection surgery evolve, adapt, and become resistant to chemotherapy and radiation treatments. They begin to grow more aggressively than the original tumor, are difficult to treat, and have limited treatment options. Likewise, when medulloblastoma spreads to the spine, called metastasis, treatment is extremely difficult as surgery and radiation/chemotherapy have immense risks. The current proposal uses our strong foundational understanding of medulloblastoma, brain development, and neuroscience to 1) advance our understanding of medulloblastoma local recurrence and 2) develop targeted therapies for patients with medulloblastoma metastases that are safe. We hypothesize that medulloblastoma will exploit a protein called GABA Transaminase, also known as ABAT, to survive in the harsh conditions caused by radiation and chemotherapy and allow medulloblastoma to recur. Moreover, because metastatic medulloblastoma are dependent on ABAT to grow, as our recent publication has shown, we hypothesize that a new potential ABAT inhibitor, NEO216, will serve as a safe and effective targeted therapy for patients. These studies will have potential near-term impact on understanding how the pediatric brain tumor medulloblastoma recurs and how we can directly target medulloblastoma metastases – the principal reasons for patient mortality. Overall, the findings from our proposed research plan will reduce the burden on patients and their families, will improve their quality of life, and most importantly, increase the overall survival of pediatric patients who suffer from this terrible disease.