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November 2005
Children's Research Institute News Brief

Dr. Maria
Bernard L. Maria, MD, MBA
Executive Director
Darby Children's Research Inst.
Inderjit Singh, PhD
Inderjit Singh, PhD
Scientific Director
Darby Children's Research Inst.


Why are Clinical and Translational Sciences critically important to young South Carolinians?
In this issue of Kids Connection, we focus on the importance of clinical and translational research to create value and health for our children. Discovery is an important component of the overall mission of the Children's Hospital. The opening of the Charles P. Darby Children's Research Institute (DCRI) in February 2005 signals a deep commitment to developing and refining new approaches and treatments for children in the future. The building itself was constructed on a beautiful historic site that reminds us daily of advances in children's care over the last 200 years. Thus, Past, Present (Children's Hospital), and Future (DCRI) have joined to meet the health needs of our children.

With all the excitement of modern science and the sophistication of medical technologies offered our patients, however, we cannot be complacent. In state-by-state comparisons of health, South Carolina often ranks poorly. The social, economic and educational components that affect the health of a society disproportionately affect our largely rural population. Recent rankings by the Centers for Disease Control and Prevention positioned South Carolina's population as follows:

Ranking & Facts

  • 1st in stroke deaths;
  • 9th in diabetes deaths;
  • 9th in prostate cancer deaths;
  • 18th in heart disease deaths;
  • 25th in cancer rates;
  • 21 percent of the population is overweight or obese.
Based on a 2000 census, one-third of South Carolina's 3.9 million residents belong to a minority group, with African Americans representing the largest group at almost 30 percent, and Hispanics a distant second at two percent of the total. Between 1990 and 2000, the Hispanic population more than doubled, and African-Americans increased by 14 percent.

All over the country, minorities experience poorer health outcomes and more premature deaths than whites. For example, in South Carolina, African-American infants are twice as likely to die before their first birthday than white babies. Paul Darden, MD, Department of Pediatrics, recently stated, "quality of life and health status are linked to both modifiable and unmodifiable factors: socioeconomic status, education, race/ethnicity, and access to care."

Of the one million children in South Carolina, approximately 138,000 do not have health insurance, more than 20 percent live in families at or below the poverty level, 26 percent of mothers get less than adequate prenatal care (leading to health risks for newborns), and 39 percent of babies are born to single mothers.

South Carolina participates in the nationwide KIDS COUNT project, funded by the Annie E. Casey Foundation, which profiles children across the nation. South Carolina ranks 44th out of the 50 states for the sum of 10 traditional measures of the well-being of children. On individual indicators directly related to health, the state ranks 46th in infant mortality, 47th in low-birth weight babies, 42nd in teen birth rate, 38th in child death rates, 42nd in teen death rates, and 34th in percent of children living in poverty. The KIDS COUNT indicators of child well-being and other data cited confirm the need for ongoing vigorous efforts to improve the health status of children and families in South Carolina through transforming clinical and translational research efforts.


What are the National Institutes of Health doing to enhancing clinical and translational sciences?
NIH Director Dr. Elias Zerhouni has made clear that the discipline of clinical and translational sciences must develop further to bridge gaps between basic science discoveries and clinical applications. There are many important challenges to the clinical research enterprise:
  • Difficulty recruiting and retaining clinical researchers.
  • Increasing regulatory burden and overhead costs.
  • Fragmented training programs.
  • Limitations and barriers due to funding mechanisms, review and program structures.
  • Explosion in clinical service demands and reduction in financial margins at academic health centers.
  • Decreased valuation attached to translational science because of the "dilution" effect due to a marked in crease in numbers of faculty
  • The complexity of knowledge needed to be an effective clinical/translational scientist.
  • The absence of a true "academic home' for young clinical faculty.
On the last bullet above, the "academic home," the National Center for Research Resources (NCRR) has just released a request for applications for Clinical and Translational Sciences Awards (CTSAs) to establish centers/institutes/departments to oversee enhancement of the discipline of clinical and translational sciences clinical and translational sciences.

They envision funding 60 centers nationally, and phasing out the 78 General Clinical Research Centers (GCRCs) through 2012. Each of the CTSAs would have career development programs (T32, K12) to develop future clinical and translational scientists. CTSAs would grow the clinical research enterprise and catalyze translation of "benchtop" discoveries to "bedside" applications (and vice-versa), and translation of clinical findings into best practices. Importantly, successful CTSAs would have to transform academic health centers and their culture. On October 26th, Drs. Kathleen Brady (new Director of GCRC) and Bernie Maria met with NCRR Director Dr. Barbara Alving to explore opportunities for MUSC. It was a wonderful opportunity to share the vision of the DCRI and how it might address NCRR and Dr. Alving's interests.

We believe that the MUSC Children's Hospital and DCRI are breaking down programmatic and disciplinary "silos" and creating new bridges across scientific fields, stimulating change at all levels. The Children's Hospital and the Department of Pediatrics have a strong track record in multidisciplinary care, education and research. One recent example of success in education that relates to the national imperative in clinical and translational sciences is the recent award of a T32 Roadmap training grant to co-PI Dr. Tom Hulsey, whose Division of Pediatric Epidemiology is featured in this issue of Kids Connection.

The DCRI has attracted investigators from 20 departments on campus to study developmental biology and children's diseases. Thus, we are building on the existing strengths of established pediatric investigators, but also creating even more value for children by accommodating other programs that are developing novel translational strategies.

As an example of pre-clinical development, Dr. Steve Tomlinson is developing targeted complement inhibitors for inflammatory conditions, Dr. Doe Eicher is working with Dr. Singh on neuroprotection in clinical trials in neonatal hypoxic ischemic encephalopathy, and Dr. Jane Charles is working with Dr. Lindsey DeVane on pharmacogenetics in autism. Dr. Paul Darden is translating discoveries into community-based research. Thus, within our world of children's research at MUSC, we have luminaries in pre-clinical, clinical, and community translational approaches that NIH is seeking for the CTSAs.

We must continue to work hard so that the DCRI can serve as a model for MUSC as a whole. It will be up to all of us to work together in coming months to make use of our pediatric values, attitudes, and experiences to leverage the needed change and to educate the greater scientific community about our models of success - as we all know, "only babies like to be changed."

As a first step, it will be up to ALL of us as clinicians, educators, and investigators to take responsibility to connect with one or more people working across the chasm. For MUSC faculty members reading this issue of Kids Connection, have you recently had a cup of coffee with a clinical or research counterpart in the Children's Hospital or DCRI to explore common interests? If not, why?


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