171 Ashley Ave.
Charleston, SC 29425
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December 2006
Children's Research Institute News Brief
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Bernard L. Maria, MD, MBA
Executive Director
Darby Children's
Research Inst. |
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Inderjit Singh, PhD
Scientific Director
Darby Children's
Research Inst. |
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Lab searches for cause and cure for lupus
Lupus is one of the most common rheumatic diseases that affect children, and Dr. Gary Gilkeson's lab in the DCRI is determined to find help for those who suffer from it.
One of two rheumatic diseases labs in the DCRI, Dr. Gilkeson's lab supports the clinical work of the state's only pediatric rheumatologists, Drs. Richard Silver and Natasha Ruth.
The lab has three major focuses, says Dr. Gilkeson, with a principal goal of controlling the gene expression of lupus.
"Our research primarily revolves around determining the genetic control of lupus," he says. The lab aims to identify genetic targets which could then be used as treatment.
"This is especially promising in children who have a higher genetic influence than adults," explains Dr. Gilkeson, a professor of medicine and vice chair of the department of medicine. Assistant professor Dr. Tammy Nowling is the principal investigator in this aspect of the lab's endeavors.
Though still in the development stage, this genetic regulation research seems poised for success, says Dr. Gilkeson.
Within sight of human trials is the lab's work with Dr. Steve Tomlinson, who has developed inhibitors of the complement pathway.
"This is an important mediator of inflammation, tissue damage and organ damage, so being able to inhibit it has important consequences," explains Dr. Gilkeson.
Drs. Tomlinson's and Gilkeson's labs have used these inhibitors to successfully treat strains of mice that have developed lupus-like diseases. "We're developing similar agents to take into human trials," says Dr. Gilkeson.
He and fellow researchers expect these complement inhibitors agents to be applicable to a variety of childhood inflammatory diseases "The first to be tried will probably be asthma, and our hopes are that they may also be effective in treating lupus and childhood arthritis.
The lab's third area of focus is the genetic control of immune deficiencies. "This research focuses on children who don't have enough antibodies, who have IgA deficiency or combined variable immune deficiency (CVID)," notes Dr. Gilkeson.
"We've identified the gene that causes this problem in mice," he says of his and assistant professor Dr. Hideharu Sekine's efforts. "This genetic identification will allow us to determine what interventions will treat or cure this deficiency."
Around the corner is new research and collaborations with new recruit Dr. Ruth. "We'll be looking at cognitive dysfunction in children with lupus, which is her area of interest," notes Dr. Gilkeson.
His lab is also busy with other rheumatic studies that involve children, including research on lupus within the Gullah population, clinical trials of new therapies that involve teens with lupus, even early screening programs for lupus in Charleston County's predominantly African-American high schools. "The hope is that we'll be able to diagnose and treat these teens early in the disease process before there is organ damage."
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