Joan & Sanford I. Weill Medical College of Cornell University Archives

2014

Scholar Grant

Dr. Praveen Raju

New York, NY

Medulloblastoma is the most common malignant brain tumor in children and a leading cause of
cancer-related death in children. More than one-third of afflicted children die within five years despite
treatment that includes surgery, whole brain and spine radiation, and chemotherapy with the surviving
patients often suffering from severe neurological deficits due to the toxicity of these intensive therapies. A
subset of medulloblastoma patients that are deemed “high risk” have been found to have medulloblastoma
cancer cells that are genetically different from each other within the tumor, suggesting that different
therapies might be needed for each of the genetically distinct cell types.

Importantly, current animal models of “high risk” medulloblastoma do not faithfully recapitulate the
situation in the human disease. To identify effective therapies that will improve outcomes for children with
“high risk” medulloblastoma, we propose to use sophisticated mouse genetics technology to develop a novel
animal model of “high risk” medulloblastoma that will allow us to assess how different cancer cells within a
medulloblastoma tumor respond to standard therapies such as radiation as well as precise targeted
therapies. Our results, if successful, will allow us to identify potential novel drug targets within the different
cancer cells that may less toxic. Finally, this novel approach may be used for generating improved animal
models for other childhood cancers in order to identify improved treatment strategies.