Pediatric CNS cancer genomics and immunogenomics

Abstract

Large-scale genomic discovery in pediatric cancers established the importance of multiple platform-based characterizations of DNA and RNA to obtain a complete molecular landscape of these cancers, including actionable variants, diagnostic or prognostic evidence, and germline susceptibility. While these discoveries set the stage for pediatric cancer precision medicine, broad-based implementation has been quite slow compared with the adult-cancer precision medicine setting, due largely to the rarity of pediatric cancer. Here, we survey several large-cohort studies that utilize multiplex clinical characterization, including pediatric patients diagnosed with central nervous system (CNS) malignancies. The reported results demonstrate that molecularly guided precision therapeutics yield clinical benefit for these patients, establishing one important component needed for precision therapeutics to enter the pediatric CNS setting.