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Last in a series on the MUSC Children's Hospital  

Research Institute leads to new discoveries

by Dick Peterson
Public Relations
Investigations into childhood diseases and birth defects appear to be a key to unlock cures for all ages.

Childhood diabetes, neuro-degenerative disorders, and heart development and defects are among the numerous multi-discipline research initiatives that will find a home in a completed Children’s Research Institute. The institute will give MUSC 49,500 square feet of research space and 10,000 square feet for office support.

“Our goal is to increase our funding level to put us in the top 25 children’s research programs in the country,” said Department of Pediatrics chairman and Children’s Hospital physician in chief L. Lyndon Key. “This is an attainable goal. We’re only limited by the space available for research.”

Among pediatrics departments nationwide, MUSC ranks 45th in federal funding for research. By comparison, the university as a whole ranks 56th, Key said. Charting his department’s annual growth, Key points to an upward trend beginning at $700,000 10 years ago and now topping $7.5 million.

“Last year was good,” Key said. “We attracted enough federal funding to put us in position to compete with most pediatrics departments in the Southeast.”

An already solid program of comprehensive pediatric clinical care and research has seen significant changes in the past two years. Key cites a team approach to delivering multi-disciplinary care to children, increased research funding and the new 121,777 square-foot Children’s Research Institute. 

A focal shift to children’s research was evident in 2001 when ground was broken for the Children’s Research Institute, now in its skeletal phase of steel I-beams and poured-concrete floors. 

Since the ground-breaking, $10 million has been raised to help complete the construction in early 2004. The institute will house a developmental and regenerative medicine laboratory dedicated to tissue development and engineering aimed at treating neurological, cardiological and diabetic disorders. 

With this initiative, the institute will draw in more than $20 million in additional research funds and attract scientists from around the world in collaborative projects. For example, a relationship with Hadassah Hospital in Israel is developing with islet cell physiologists.

In conjunction with basic research efforts, MUSC’s Children’s Hospital is about to complete construction of a cellular isolation facility to produce islet cells and other cellular tissues for use in transplantation programs. 

“We are developing an islet cell transplantation program backed by a program in adult stem cell biology in an attempt to cure children with diabetes,” Key said. Under his direction, the work focuses on moving transplantation technology into younger patients and involves specialists from the departments of Pathology, Surgery, Radiology, Pediatrics and Medicine.

Research into childhood disease processes, genetic disorders, and trauma by Distinguished University Professor Inderjit Singh, Ph.D., demonstrates the value of pediatric-related investigation to both children and adults. A study of childhood diseases and how they relate to adult inflammatory diseases, led Singh to document the efficacy of statins in animal models of multiple sclerosis, stroke and Alzheimer’s disease. The statin, Zocor, a cholesterol-reducing drug, is in human trials to test its ability to prevent the relapsing-remitting form of multiple sclerosis with preliminary results showing promise.

Another Singh project in collaboration with MUSC’s transplant program studies the beneficial effect of anti-inflammatory drugs in renal ischemic injury. “These approaches led to improvements in organ preservation,” Singh said. “Now a kidney can be stored for up to 48 hours in newly developed solutions and still function normally after transplantation.”

Singh said that although his initial studies have used kidneys only, he expects the approach to increase the availability of other organs for transplantation. “Human clinical trials based on these findings are presently in the planning stages,” Singh said.

Basic research into heart development will occupy half of the sixth floor of the institute connected to the floor that research now occupies in the Basic Science Building, said J. Philip Saul, M.D., director of pediatric cardiology. “The research investigates factors that make the heart form normally or cause defects,” he said. 

Conducted in the Department of Cell Biology and Anatomy under Roger Markwald, Ph.D., the research centers on stem cells, genes, and proteins that control cardiac development and molecules that predispose a heart to develop abnormally. 

In clinical research, the institute participates in a number of multi-center studies—studies that require a larger population of patients than one institution could provide. Among them is a study in Kawasaki disease, a childhood disease that primarily affects children under 5 years old and is the leading cause of acquired heart disease in children. 

Another study examines factors affecting outcomes from the Fontan procedure, a surgical technique used for people who have only one effective heart pumping chamber instead of the usual two. There are drug studies and catheter ablation trials to correct heart rhythm abnormalities. And “MUSC is one of the top centers to implant investigational devices for repairing heart defects,” Saul said.

Other projects under the aegis of the Children’s Hospital and Children’s Research Institute include research in hematology and oncology, sickle cell disease and reasons for blood cell occlusion, strokes of the brain, osteoporosis, the role of vitamin D in bone density and rickets, autism, the prevention of underage pregnancy, and an epidemiological approach to factors affecting the health of newborns.
 
 

Catalyst Online is published weekly, updated as needed and improved from time to time by the MUSC Office of Public Relations for the faculty, employees and students of the Medical University of South Carolina. Catalyst Online editor, Kim Draughn, can be reached at 792-4107 or by email, catalyst@musc.edu. Editorial copy can be submitted to Catalyst Online and to The Catalyst in print by fax, 792-6723, or by email to petersnd@musc.edu or catalyst@musc.edu. To place an ad in The Catalyst hardcopy, call Community Press at 849-1778.