Molecular Pathogenesis and Emerging Treatment for Glioblastoma

Glioblastoma is the most common primary malignant brain tumor, with more than10,000 new cases each year in the United States. Significant basic science and clinical research has been devoted to understanding this disease, yet median survival with standard of care treatment remains approximately 15 months. Over the past decade, advances in genomic sequencing technology, biostatistics, and computing have allowed for an unprecedented ability to profile the gene expression patterns and mutations driving the formation of tumors. These advances have generated both prognostic information as well as a multitude of treatment targets that are just now coming into clinical practice. This article aims to provide a comprehensive update on the recent use of genetic profiling to identify the molecular pathways altered in glioblastoma and to describe ongoing clinical trials to exploit these pathways for treatment.