Personalized Medicine in Pediatric Cancer

The maturation of genomic technologies has enabled new discoveries in disease pathogenesis as well as new approaches to patient care. For most of its 70-year history, systemic cancer treatment has relied on drugs marginally more toxic to malignant cells than to normal tissues. Molecular markers to predict benefit or understand therapeutic resistance in the clinic have usually been lacking. In pediatric oncology, patients may now receive individualized genomic analysis to identify molecular aberrations of relevance for diagnosis and/or treatment. Several recent clinical studies have begun to explore the feasibility and utility of genomics-driven precision medicine. Precision medicine is broadly defined by the National Institutes of Health as an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person. We review the major developments in this field, discuss current limitations, and explore aspects of the clinical implementation of precision medicine, which lack consensus. Leading international researchers and pediatric cancer advocates will discuss opportunities and gaps in the research in 4 main tumor types: Brain cancers, Leukemia, Embryonal tumors, Sarcomas. We discuss ongoing scientific efforts in this arena, which may yield future clinical applications. Precision medicine in pediatric oncology drug development: the right to accelerate innovation for children and adolescents with cancer and work together and re-invent partnerships. The goals of precision medicine in pediatric oncology increase genomics-based clinical and preclinical studies of cancer treatment: Expand genomics-based clinical trials, understand & overcome resistance to targeted drugs; drug combinations and build repository of patient-derived pre-clinical models for evaluating targeted therapeutics, create national cancer database to integrate genomic information with clinical response and outcome.