Medulloblastoma: From Molecular Subgroups to Molecular Targeted Therapies

Annu Rev Neurosci. 2018 Jul 8;41:207-232. doi: 10.1146/annurev-neuro-070815-013838. Epub 2018 Apr 11.


Brain tumors are the leading cause of cancer-related death in children, and medulloblastoma (MB) is the most common malignant pediatric brain tumor. Advances in surgery, radiation, and chemotherapy have improved the survival of MB patients. But despite these advances, 25-30% of patients still die from the disease, and survivors suffer severe long-term side effects from the aggressive therapies they receive. Although MB is often considered a single disease, molecular profiling has revealed a significant degree of heterogeneity, and there is a growing consensus that MB consists of multiple subgroups with distinct driver mutations, cells of origin, and prognosis. Here, we review recent progress in MB research, with a focus on the genes and pathways that drive tumorigenesis, the animal models that have been developed to study tumor biology, and the advances in conventional and targeted therapy.

Keywords: animal models; medulloblastoma; molecular subgroups; targeted therapy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cerebellar Neoplasms* / classification
  • Cerebellar Neoplasms* / genetics
  • Cerebellar Neoplasms* / therapy
  • Humans
  • Medulloblastoma* / classification
  • Medulloblastoma* / genetics
  • Medulloblastoma* / therapy
  • Molecular Targeted Therapy / methods*