The Dawn of Pediatric Personalized Therapeutics

Abstract

Address.correspondence.to.Stephen.P..Spielberg,.MD,.PhD,. Marion.Merrell.Dow.Chair.in.Pediatric.Pharmacogenomics;. Director,.Center.for.Personalized.Medicine.and.Therapeutic. Innovation,.Children’s.Mercy.Hospital,.2401.Gillham. Road,.Kansas.City,.MO.64108,.email:.sspielberg@cmh.edu © 2010 Pediatric Pharmacy Advocacy Group It is a great honor for me to have been awarded the Sumner Yaffe Lifetime Achievement Award in Pediatric Clinical Pharmacology. Dr. Yaffe has been a lifelong mentor and colleague, a true definer of a discipline, creator of the Pediatric Pharmacology Research Units (PPRU) sponsored by the NICHD, and a dear friend. I am tremendously grateful to the PPAG for honoring me with this award in his name. The last years have been important ones for pediatric therapeutics. The scientific basis has been driven by huge advances in pediatric clinical pharmacology, fundamental understanding of the ontogeny of drug disposition and of drug response. United States legislation including the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act have changed the drug development process, leading to new knowledge about the safe, effective use of medicines in children. Research networks including the Pediatric Pharmacology Research Units (PPRUs), the Children’s Oncology Group, and now the Clinical and Translational Science Award (CTSA) network have increased pediatric investigative activities throughout the country. The National Institutes of Health (NIH) and the Food and Drug Administration (FDA) have ABBREVIATIONS ADRs,.adverse.drug. reactions;.CTSA,. Clinical. and.Translational. Science. Award;. CYP2D6,. Cytochrome.P2D6.System;.CYP2D9,.Cytochrome.P2D9. System;.EMEA,.European.Medicines.Evaluation.Agency;. FDA,.Food.and.Drug.Administration;.GENDEP,.Genome. Based.Therapeutic.Drugs. for.Depression;.GWAS,.whole. genome.association. studies;.MDD,.major. depressive. disorder;.NICHD,.National. Institute.of.Child.Health.and. Human.Development;.NIH,.National.Institutes.of.Health;. PPRU,. Pediatric. Pharmacology.Research.Units;. SSRIs,. selective.serotonin.reuptake.inhibitors. both played critical roles, with the FDA Critical Path initiative in many ways reflecting scientific thinking about translational medicine driving