Review

Neurosurgical Review

, 31:359

First online:

The molecular genetics of medulloblastoma: an assessment of new therapeutic targets

  • Carlos G. Carlotti JrAffiliated withDivision of Neurosurgery, Ribeirão Preto Medical School, University of São Paulo
  • , Christian SmithAffiliated withThe Division of Neurosurgery, Suite 1503, The Hospital for Sick Children, and The Arthur and Sonia Labatt Brain Tumour Research Centre, The University of Toronto
  • , James T. RutkaAffiliated withThe Division of Neurosurgery, Suite 1503, The Hospital for Sick Children, and The Arthur and Sonia Labatt Brain Tumour Research Centre, The University of Toronto Email author 

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Abstract

Medulloblastoma is the most common pediatric primary malignant intracranial neoplasm. The 5-year survival rates vary from 40% to 70% depending on clinical prognostic criteria, and many of the patients who survive exhibit long-term neurocognitive and/or neuroendocrine sequelae. Because of these results, research is required to increase our understanding of the basic biology of medulloblastoma, helping to refine patient stratification, decrease side effects of treatments, identify novel prognostic markers, and discover new less toxic therapies. The recognition that some medulloblastomas occur in familial cancer syndromes has led to some important discoveries in the molecular pathogenesis of medulloblastoma. These syndromes provide us with clues regarding alterations in key signaling or growth factor activation pathways that contribute to medulloblastoma formation. A better understanding of the molecular pathways involved in medulloblastoma formation may allow the discovery of new drugs that act on specific targets, yet many steps must still be taken before clinical use of new drugs. In addition, the identification of a novel signaling pathways in medulloblastoma is often accompanied by the quest for novel pharmacotherapeutics that have the potential to act favorably on this disease.

Keywords

Medulloblastoma Familial cancer syndromes Hedgehog pathway Wnt pathway Notch pathway Cyclopamine