The CPPT research efforts are quite diverse. Unlike basic science Centers with a narrow programmatic focus, the CPPT investigators touch upon the wide range of pediatric pharmacotherapeutic questions. The CPPT has advanced research in the areas of pediatric pharmacogenetics, pharmacogenomics, pharmacokinetics, pharmacodynamics, molecular pharmacology, renal therapeutics, nutrition, endocrinology, critical care medicine, gastroenterology, infectious diseases, pulmonology, and neurology in the last five years. It is likely that the Center will continue to impact a wide variety of related fields of research, education, and practice.
The CPPT continues to meet its research objectives, however there is always more discovery ahead. This work includes determining the relationship between patient characteristics (genetic, biochemical, clinical, etc.), and discrete features of drug distribution in children, and by basic research efforts aimed at characterizing the maturation of drug distribution processes and drug effect at its site of action. A coordinated but diverse clinical and laboratory research program has been carried out to characterize the determinants of drug absorption, distribution, metabolism, and elimination, and define the relationship of drug concentration to drug effect in children. The relation of these processes to measurable patient characteristics has been used to develop biomedical models for determining optimal pediatric drug dosing strategies. This has been accomplished for a number of individual drug classes (i.e. chemotherapeutic, antibiotic, antihypertensive, nutritional, and more), and new prospective assessment of dosing strategies has been conducted to evaluate their accuracy for achieving target serum drug concentrations, their ability to increase therapeutic efficacy and/or reduce toxicity, and finally their impact on cost-benefit of drug therapy in children. A CORE laboratory facility supports drug, metabolism and nutrition, and pharmacokinetic and pharmacogenetics analyses; all shared by Core and Participating faculty of the CPPT. Basic research approaches have been conducted to support clinical research finding (e.g., determine the rate at which metabolic processes mature and identify biological factors that influence the rate and extent of maturation).
The CPPT-supported Pediatric Pharmacology Research Unit is a federal program promoting increased medication labeling for children. Center-based studies have expanded our understanding of safe and effective use of medication in children. The Center has helped in securing University funding for the Center for Functional Genomics, Dr. Chesney serving as co-director. Both Centers are dedicated to better understanding of how individual and population genotypic and phenotypic expression impact drug disposition, dosing, and selection, and, importantly, how it may impact drug design and development.
The Center, in partnership with the University of Chicago, is one of 6 centers funded as part of the National Pharmacogenetic Research Network. This 11 million dollar, 4-year award from NIH is supporting expanding research to evaluate the inherited basis of individual difference in response to anticancer drugs in children.