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.
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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.