Abstract

Central nervous system embryonal brain tumors comprise a heterogeneous group which includes medulloblastoma (MB), central nervous system primitive neuroectodermal tumors (CNS-PNETs), and pineoblastoma. They are highly aggressive malignant tumors that often arise in children, are difficult to treat, and cause significant cancer-related morbidity or mortality. There has been tremendous gain in the survival of localized MB in recent years. However, treatment remains highly toxic and punishing and is much less effective for metastatic MB and non-MB PNET while recurrent MB remains largely incurable—underscoring the need to better define diagnostic and therapeutic approaches to this wide spectrum of biological diseases that receive similar multimodal therapeutic regimens. Global genomic studies have now separated embryonal tumors into clinically relevant molecular classes and are paving the way for a new era of biology-informed clinical management of these tumors. This chapter will review current clinical understanding of MB, CNS-PNET, and pineoblastoma and insights into novel therapeutic approaches for these diseases.

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