Data-driven approaches to advance research and clinical care for pediatric cancer

Pediatric cancer is a rare disease with a distinct etiology and mutational landscape compared with adult cancer. Multi-omics profiling of retrospective and prospective cohorts coupled with innovative computational analysis have been instrumental in uncovering mechanisms of tumorigenesis and drug resistance that are now informing pediatric cancer clinical therapy. In this review we present the major data resources of pediatric cancer and actionable insights into pediatric cancer etiology stemming from the identification of oncogenic gene fusions, mutational signature analysis, systems biology, cancer predisposition and survivorship studies - that have led to improved clinical diagnosis, discovery of new drug-targets, pharmacological therapy, and screening for genetic predisposition. Ultimately, integration of large-scale omics datasets generated through international collaboration is required to maximize the power of data-driven approaches to advance pediatric cancer research informing clinical therapy.