Targeted therapy in medulloblastoma in molecularly targeted therapy for childhood cancer

Medulloblastomas are primitive embryonal tumors of the central nervous system arising exclusively in the cerebellum. They have a tendency to disseminate via CSF spaces throughout the brain and spine and are the most common malignant brain tumors in children (CBTRUS 2005). There has been considerable progress in our understanding of the mechanisms driving these tumors through the study of normal cerebellar development, familial cancer syndromes, genome-based analyses of human primary tumor samples (Table 1) and genetic mouse models. These studies have provided a framework for the development of several targeted therapies. This chapter focuses on our current molecular understanding of medulloblastoma and the targeted therapies in development and clinical trials.