Pre-clinical Testing of Novel Glutamine Metabolic Inhibitors in MYC-driven Medulloblastoma
Medulloblastoma is the most common pediatric brain tumor. While up to 80% of patients are cured, there is a subgroup of patients for whom the current aggressive therapies do not work. These patients have tumors which make large amounts of a protein called MYC. This is a very strong driver of cancer cell growth, but MYC is very hard to target directly.
MYC is known to change the metabolism of cancer cells and this proposal will develop new drugs specifically targeting the metabolic changes downstream of MYC. We have already found that one of these drugs, called DON, when given to lab mice will slow the growth of medulloblastoma tumors. We will modify DON to make it easy to take in an oral formulation. We will also alter DON so that it gets into the brain better. Support from Alex's Lemonade Stand will help us develop new drugs for children with MYC-driven medulloblastoma who currently have a low chance of surviving.