Medulloblastoma is the most common malignant primary brain tumor in children. Standard of care treatment consists of surgical removal of the tumor followed by chemotherapy and radiation, depending on the patient’s staging and age. Radiation therapy is rarely administered to children under three years of age because of the high potential for serious neurodevelopmental deficits, further limiting the treatment options available to the youngest patients. While modern treatments have led to improvements in survival, the risk of recurrence as well as the long-term side effects associated with surgery, radiation, and chemotherapy underscore the urgent need for new therapeutic approaches.