EXCEED THE SPACEPROVIDED.This proposal is for the continuation of support for the 6-bed General Clinical Research Center (GCRC), itscore laboratory and its outpatient research program at the Children's Hospital, Boston, the largest medicalcenter in New England devoted to the care of children and the principal pediatric program affiliated withHarvard Medical School. Children's Hospital has 325 beds, 17,000 inpatient admissions, and 350,000outpatient visits annually. This GCRC has a long history of clinical research innovation exemplified by the1996 Award for Excellence in Clinical Research bestowed upon Dr. David G. Nathan, then PrincipalInvestigator. The central mission of this GCRC is to integrate basic and clinical research, to elucidatemechanisms of disease, to formulate novel therapies and to facilitate their translation to clinical care. Ouraccomplishments follow this paradigm. In the past few years a pilot study of the administration of high dosecorticosteroids to patients with Kawaski disease demonstrated improvement in a number of outcomesleading to a nationwide NIH funded clinical trial led by one of our investigators. Investigators characterizingthe biology of ion channels have contributed to studies in sickle cell and inflammatory bowel diseases.Observations in patients with adenomas associated with glycogen storage disease, a long supported GCRCstudy, led to the proposed investigations into the role of hepcidin in regulating the anemia of chronic disease.Studies on the control of insulin in patients taking high and low glycemic index diets have led to a new NIHfunded trial by this investigator. The investigation of infants appearing in our emergency room withhypoglycemia established that disorders in fatty acid metabolism occur in a substantial number, and shouldchange management of such children. The Principal Investigator who is also the President and CEO of thehospital has succeeded in expanding participation of all departments in GCRC research. Among the newinitiatives will be the development of a Phenotype-Genotype Core designed to extend understanding of thegenetics and genetic modifiers of pediatric diseases, and the expansion of the Nutrition-Metabolism Core todevelop novel therapies in response to the epidemic of obesity among children and adolescents. Substantialefforts and resources from the hospital will be directed toward attracting young investigators into clinicalresearch. Overall, the GCRC at Children's Hospital has both initiated and responded to clinical researchdemands across the institution.
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