Evaluating inhibitors of exosomes secretion as pan-treatment for medulloblastoma

Significance: Medulloblastoma is the most common malignant pediatric brain tumor. Current therapy, developed empirically more than 50 years ago, follows surgical removal with the nonspecific regimen of radiation and chemotherapy. This treatment yields 70-80% long-term survival, but survivors suffer disabling long-term toxicities, including dementia, early strokes, and hearing loss. (1) Patients that survive remain at risk of recurrence, which is presently incurable, and up to 60% of medulloblastoma patients depending on the subgroup eventually die of disease (2-4). Therefore, there is an intense interest in developing effective and less toxic therapies to improve clinical outcomes for children affected by medulloblastoma. However, the majority of treatments remain subgroup-specific due to different driving pathways.