Molecular Biology of Pediatric Brain Tumors and Impact on Novel Therapies

Brain tumors are the leading cause of cancer-related death in children. For the past several decades, therapeutic strategies have centered on cytotoxic chemotherapy and radiation therapy due, in part, to limited understanding of genetic events that underlie tumor initiation and maintenance. Significant improvement in high-throughput genomic methods, such as next-generation sequencing, methylation array, and copy number array, in recent years has propelled the knowledge base from which novel therapies are derived. Translation of recent genomic findings into more effective therapies remains the most formidable challenge in improving the outcome for children with brain tumors.