The Research Institute at Nationwide Children’s Hospital has made tremendous strides in recent years. More than quadrupling its size, it houses 13 Centers of Emphasis where more than 100 scientists and physician researchers work around the clock to search for new treatments and cures for chronic and deadly diseases. Ranked among the top 10 free-standing pediatric research facilities in the United States according to National Institutes of Health funding, The Research Institute laboratories are also where more than 100 research fellows and graduate students train annually. Because of this growth, The Research Institute will have its own Update page in every issue of Pediatric Directions.
Kaspar’s Research Findings Published in Nature Magazine
Brian Kaspar, principal investigator at The Research Institute at Nationwide Children’s Hospital had his research findings published in Nature Biotechnology, January can shuttle genes across the blood–brain barrier. The discovery raises hopes that gene therapy could one day be used to treat neurodegenerative diseases. Although a variety of viruses have been harnessed as vectors to carry genes into cells, none has been able to enter the brain that adeno-associated-virus 9 (AAV9) carrying a fluorescent ‘reporter’ gene enters neurons in the brain and spinal cord when injected intravenously into newborn mouse pups, in which the blood-brain barrier is not fully formed. AAV also entered the central nervous system of adult mice, but primarily targeted support cells called astrocytes.
Gail Besner, MD, Receives John E. Fisher Endowed Chair in Neonatal Research
Gail Besner, MD, was presented with the John E. Fisher Endowed Chair in Neonatal Research, December of 2008. Dr. Besner is a Professor of Surgery and Pediatrics at The Ohio State University College of Medicine, and is the Burn Director and Associate Program Director of the Pediatric Surgery Residency Training Program at Nationwide Children’s Hospital. She is also a principal investigator in the Center for Perinatal Research housed in the hospital’s research institute. Dr. Besner’s research focuses on a growth factor known as heparin-binding EGF growth factor, or HB-EGF, which she discovered in 1990. The translational component of her research involves neonatal necrotizing enterocolitis, or NEC, which results in severe intestinal injury in newborns, especially those born prematurely, often leading to death or severe disability of the newborn. Her goal is to develop therapeutic regimens for high-risk newborns, using HB-EGF to prevent and treat this devastating disease. Dr. Besner currently holds three separate million-dollar NIH grants to fund her research. The endowed chair is the fifth and final chair funded in part by the $50 million contribution to Nationwide Children’s from the Nationwide Foundation. The chair is named for John E. Fisher, who served as General Chairman and CEO of Nationwide Insurance Companies from 1981 to 1992. He retired as General Chairman in 1994, and passed away in 1998.
Nationwide Children’s Elected Leader of Cystic Fibrosis Translational Research
A new designation will allow Nationwide Children’s Hospital to lead cutting-edge research combating the life-shortening, genetic disease cystic fibrosis. Among 116 specialized cystic fibrosis centers throughout the country, Nationwide Children’s has been selected to serve as one of 13 translational research centers for the Cystic Fibrosis Foundation’s Therapeutics Development Network (CFF TDN). These translational research centers will lead the newest Phase I clinical trials and provide scientific direction to the entire network. Historically, the CFF TDN has been comprised of 18 clinical research centers intended to fast-track improvements in cystic fibrosis care by streamlining the research in an environment of patient safety and high standards of research consistency. In order to meet the growing need for study participants and expanded research programs, a decision was made in 2007 to expand the network to include 64 “routine research centers” and 13 translational research centers. The Section of Pulmonary Medicine at Nationwide Children’s participated in its first cystic fibrosis clinical trial in 1992, the third-ever cystic fibrosis clinical trial world-wide, and has since taken part in more than 50 clinical trials for cystic fibrosis. The hospital has been a CFF TDN member since 2002.