Children's receives one of only two national pediatric rheumatology training grants

09/08/05

Children's Hosptial of Pittsburgh leads the way in training tomorrow's pediatric rheumatologists - which is crucial to the survival of experts in the field

Children's Hospital of Pittsburgh's Rheumatology Division received one of only two National Institutes of Health (NIH) training grants and is recognized as one of the foremost programs devoted to children with rheumatic diseases in the United States.

Children's was awarded a five-year grant from the NIH to train fellows in pediatric rheumatology. Under the leadership of Raphael Hirsch, MD, division chief of Pediatric Rheumatology at Children's, the program will receive funding for training four fellows a year (two second-year fellows and two third-year fellows).

Dr. Hirsch said the NIH-funded grant is critical to the survival of experts in the field of pediatric Rheumatology. Since there are a very limited number of certified pediatric rheumatologists in the United States, without properly training young medical professionals, the number of specialists will continue to decrease.

According to the American Board of Pediatrics, there are only 207 certified pediatric Rheumatologist in the United States. There are 6 full-time faculty, and 22 staff, in the Rheumatology division at Children's Hospital of Pittsburgh. Many states do not have a pediatric rheumatology program and are without a pediatric rheumatologist.

"There is a tremendous need for this because pediatric rheumatology is one of the most under-represented pediatric specialties, with only about 20 fellows a year in training nationwide," said Dr. Hirsch. "With this grant, Children's will have a major impact on the training of pediatric rheumatologists."

Children's provides comprehensive, family-centered care to children, adolescents and young adults with rheumatic diseases. As a leading researcher in pediatric rheumatology, Dr. Hirsch labors in a field desperate for like-minded scientists and answers to the most fundamental questions about juvenile rheumatoid arthritis (JRA) and other rheumatic diseases. Dr. Hirsch's NIH-funded laboratory is one of the few dedicated to finding what causes pediatric rheumatic diseases and how to cure them.

Dr. Hirsch and his team are using DNA micro arrays, or "DNA chips," to examine the gene profiles of both mice with induced arthritis and JRA patients to identify the genetic characteristics of the disease. Of particular importance is learning which genes are being over-expressed and which are being under-expressed in a particular patient at a specific point in time.

These gene expression profiles will help identify new targets for therapy and might someday help doctors more accurately diagnose JRA, predict outcomes and even determine which therapies individual patients are most likely to respond to. Gene expression analysis has already led to the discovery of several genes that appear to be involved in the destruction of the joint itself.

In another project, delivering gene therapies to diseased synovium of children with JRA is being investigated to improve the safety and effectiveness of new biologic drugs -- such as etanercept -- that dramatically ease disease symptoms.

"The concept is that the gene becomes the drug," Dr. Hirsch says. "We are delivering the gene to the synovium itself. Synovial cells then incorporate it into their genetic machinery and begin to make the protein locally."

The goal is to achieve steady-state levels of short-lived biologic agents in the diseased joints of patients and to eliminate systemic delivery. This, in turn, lessens the risk of possible side effects associated with the tumor necrosis factor-blocking drugs, whose long-term effects on the body's tumor surveillance capabilities is unclear.

Not only is Dr. Hirsch searching for the cause of JRA in the laboratory, he also is recruiting some of the best researchers and clinicians to join him in paving the way to more effective therapies.

"Although we are interested in the basic biology," Dr. Hirsch says, "everything we do is really focused on how to apply that understanding toward bettering the outcomes of our patients."

Source: Eurekalert & others

Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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