Molecular Genetics of Medulloblastoma in Children: Diagnostic, Therapeutic and Prognostic Implications

Abstract

Medulloblastoma is the most common embryonal tumor in children. The current standard of care comprises surgical resection, radiation and chemotherapy. Patients are stratified into standard and high risk based on the degree of resection, presence of metastatic disease and histopathology. Cure rates dramatically improved during the past decades reaching 70–80% (high and average risk, respectively). Infant medulloblastoma has a worse outcome as the use of radiation therapy is very limited, a group of patients still has dismal outcome despite appropriate therapy, and the unacceptable long-term therapy side effects in survivors. Advanced molecular techniques have allowed scientists to discover four distinct molecular subgroups and correlate them with multiple factors such as histopathology, clinical behavior and possible therapeutic targets.