Aggressive therapies – including surgery, radiation and high-dose chemotherapy – have improved outcomes for medulloblastoma patients, but many patients still die of their disease, and survivors suffer severe long-term side effects from therapy.
Most medulloblastoma research has focused on the primary tumors growing in the cerebellum. However, most medulloblastoma patients do not die from the primary disease, but from leptomeningeal metastasis: the dissemination of tumor cells from the cerebellum into the brain and spinal cord.
Genomic analyses have revealed that medulloblastoma is an extremely heterogeneous disease, with at least 4 distinct subtypes that differ in terms of mutations, gene expression, epigenetic changes, and patient survival.
Immunotherapy is emerging as a powerful approach to treating cancer. Antagonists of immune checkpoint regulators, T lymphocytes engineered to recognize tumor antigens, and vaccines that amplify tumor-specific lymphocytes are being tested against a variety of human malignancies.