title: Translating genomic discoveries to the clinic in pediatric oncology.
abstract: OBJECTIVE
  The present study describes the recent advances in the identification of targetable genomic alterations in pediatric cancers, along with the progress and associated challenges in translating these findings into therapeutic benefit.
  
  
  RESULTS
  Each field within pediatric cancer has rapidly and comprehensively begun to define genomic targets in tumors that potentially can improve the clinical outcome of patients, including hematologic malignancies (leukemia and lymphoma), solid malignancies (neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, and osteosarcoma), and brain tumors (gliomas, ependymomas, and medulloblastomas). Although each tumor has specific and sometimes overlapping genomic targets, the translation to the clinic of new targeted trials and precision medicine protocols is still in its infancy. The first clinical tumor profiling studies in pediatric oncology have demonstrated feasibility and patient enthusiasm for the personalized medicine paradigm, but have yet to demonstrate clinical utility. Complexities influencing implementation include rapidly evolving sequencing technologies, tumor heterogeneity, and lack of access to targeted therapies. The return of incidental findings from the germline also remains a challenge, with evolving policy statements and accepted standards.
  
  
  CONCLUSIONS
  The translation of genomic discoveries to the clinic in pediatric oncology continues to move forward at a brisk pace. Early adoption of genomics for tumor classification, risk stratification, and initial trials of targeted therapeutic agents has led to powerful results. As our experience grows in the integration of genomic and clinical medicine, the outcome for children with cancer should continue to improve.
authors.affiliation: aDivision of Pediatric Hematology/Oncology and Stem Cell Transplantation, Department of Pediatrics, Columbia University Medical Center, New York City, New York bDivision of Pediatric Hematology/Oncology, Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA.
authors.forename: Julia
authors.initials: J
authors.lastname: Glade Bender
authors.forename: Anupam
authors.initials: A
authors.lastname: Verma
authors.forename: Joshua D
authors.initials: JD
authors.lastname: Schiffman
mesh_terms: D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D006801:Humans; D033162:Incidental Findings; D007223:Infant; D008495:Medical Oncology / Q000639:trends*; D058990:Molecular Targeted Therapy / Q000639:trends*; D009369:Neoplasms / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D057285:Precision Medicine / Q000639:trends*; D057170:Translational Research, Biomedical / Q000379:methods / Q000639:trends