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Department of Pediatrics   

Michael Brady, MD, Chair

Pediatrics faculty are based at Nationwide Children’s Hospital. Their research is organized into multidisciplinary Centers of Emphasis and overseen by The Research Institute, which enhances the health of children through research. Centers of Emphasis consist of four to 12 faculty members and include programs in biobehavioral health, cardiovascular medicine, cell and developmental biology, cancer, clinical and translational research, gene therapy, injury research and policy, innovation in pediatric practice, mathematical medicine, molecular and human genetics, microbial pathogenesis, perinatal research, and vaccines and immunity. National Institutes of Health (NIH) funding to The Research Institute reached $24 million in 2008. Total external awards were $49 million, placing Nationwide Children’s in the top 10 in NIH dollars received by freestanding children’s hospitals. Faculty published more than 500 works in 2008. A major initiative was to enhance clinical and translational research at the hospital and in partnership with Ohio State by participating in the University’s Clinical and Translational Science Award application. This award was granted in May 2008.

Ongoing Research Programs

  • Lauren Bakaletz, PhD, directs the Center for Microbial Pathogenesis. In her research program, a national leader in the study of innate immune mechanisms relevant to otitis media, a long-term goal is to develop methods to treat or prevent otitis media caused by non-typeable Haemophilus influenzae. Promising preclinical vaccine trials are under way.
  • Jerry Mendell, MD, is a neurologist and director of the Center for Gene Therapy. Mendell and collaborators have assembled a sophisticated clinical and translational research program aimed at treating Duchenne muscular dystrophy by novel methods, including adeno-associated viral (AAV)-based gene therapy. Phase 1 clinical trials are under way.
  • Chris Walker, PhD, director of the Center for Vaccines and Immunity, is a national leader in research on the immunopathogenesis of hepatitis C, a major human pathogen that may be acquired perinatally. A major focus is understanding how immune responses are subverted in hepatitis C, leading to persistent virus infection of the liver. Collaborative research projects on perinatal acquisition of hepatitis C are under way with the Department of Obstetrics and Gynecology.
  • The research group led by Gary Smith, MD, DrPH, in the Center for Injury Research and Policy, strives to increase scientific understanding of the epidemiology, prevention, treatment, rehabilitation and biomechanics of injuries to children. Smith’s group was designated as an official Centers for Disease Control and Prevention Injury Control Research Center in 2008.
  • Investigators in the Center for Innovation in Pediatric Practice, led by Kelly Kelleher, MD, MPH, are studying techniques to improve the health of children and their families through research on novel methods of delivering health services in the community. These methods include computer and information technologies as well as innovative communication techniques.

Research Accomplishments of 2008

  • Brian Kaspar, PhD, and colleagues in the Center for Gene Therapy published their ongoing work on gene therapeutic approaches for muscular dystrophy in the journal Proceedings of the National Academy of Sciences, USA. The team used a single dose of intramuscular adeno-associated virus vector housing the myostatin-inhibitor follistatin to assess its effects on muscle mass and function. Increased muscle mass and strength were observed in wild type controls as well as mdx mice, a model of human muscular dystrophy; this study advanced the group’s progress in designing molecular approaches for treatment of neuromuscular disorders such as muscular dystrophy.
  • Christine Collins and Dawn Comstock, PhD, investigators in the Center for Injury Research and Policy, studied epidemiological aspects of injuries in high school baseball players and published their findings in the journal Pediatrics. An innovative feature of this research was the use of Reporting Injuries Online (RIO), a national Web-based high school injury reporting tool invented by Comstock. Using RIO, the investigators analyzed 131,555 high school baseball injuries and found that head/face and mouth/teeth injuries for pitchers, infielders and batters were more common than expected, leading to speculation that these injuries could be prevented with additional equipment protection.
  • Kim McBride, MD, and his colleagues in the Center for Molecular and Human Genetics studied the genetics of left ventricular outflow tract (LVOT) anomalies in 91 children with congenital heart disease, including congenital aortic stenosis, aortic coarctation and hypoplastic left heart syndrome. Reporting in the journal Human Molecular Genetics, the researchers said NOTCH mutations were found in 15.4 percent vs. 3.9 percent of controls and noted that these mutations affected NOTCH signaling in in vitro assays. This study further supports a genetic origin for many cases of congenital heart disease.
  • Jeff Bridge, PhD, and his colleagues in the Center for Innovation in Pediatric Practice explored suicide trends in adolescents in the United States using a national database. Significant excess mortality due to youth suicide was found during 2004-2005, suggesting that a sustained trend that began the year earlier was not a single-year anomaly. This study, published in the Journal of the American Medical Association, was a clarion call for more investigative work to understand the origin of this disturbing trend.
  • Faculty in the Division of Neurology made major contributions to the study of pediatric stroke, an important cause of chronic morbidity in children. Steve Roach, MD, was the lead author on a highly visible American Heart Association Scientific Statement that appeared in the journal Stroke on management of stroke in infants and children. Warren Lo, MD, led a multidisciplinary team – including faculty from the Division of Neurology, the Division of Hematology, the Center for Innovation in Pediatric Practice, and the Center for Biobehavioral Health – in publishing in Stroke the first-ever study of the costs of stroke in children.
  • The Research Institute at Nationwide Children’s Hospital hired Chris Willson, PhD, as director of technology commercialization. Willson works in concert with the Office for Technology Licensing and Commercialization at Ohio State. Technology-transfer activity in 2008 included 19 invention disclosures, two patents, one license agreement and one option agreement.