Medulloblastoma: Clinical Challenges and Emerging Molecular Discoveries

Medulloblastoma is the most common type of malignant brain tumor in children, responsible for 25% of pediatric brain cancers. Conventional treatment methods, which include surgery, radiotherapy, and chemotherapy, have improved overall survival rates for patients with medulloblastoma to over 50%. A majority of survivors, however, suffer serious long-term side effects, including developmental, neurological, and psychoso‐ cial deficits. Now entering clinical trials for sonic hedgehog-driven medulloblastomas, Smoothened inhibitors have been FDA approved for the treatment of basal cell carcino‐ mas. However, treatment efficacy endures only for a few months before lesion relapses and drug resistance occurs. Therefore, there is an urgent need for new therapies to reduce the significant problems associated with current drug-resistant treatments. In this chapter, we will illustrate the clinical presentation and current treatment methods for medullo‐ blastoma and detail the molecular pathways within each of the four molecular sub‐ groups of medulloblastoma, with an eye for possible candidates for novel combination therapies.