PITTSBURGH, March 16 -- The field of gene therapy has transformed dramatically, moving from its anticipated primary application of treating genetic disorders to managing chronic illnesses. The University of Pittsburgh Medical Center (UPMC) is at the forefront of gene therapy research for a variety of musculoskeletal disorders including arthritis, osteoporosis, muscle strains and osteogenesis imperfecta (OI). Several UPMC scientists are presenting their findings at the 44th annual Orthopaedic Research Society (ORS) meeting being held March 16 - 19 in New Orleans.
Among the most exciting in terms of clinical application includes a progress report on the first human trial for arthritis gene therapy. Rheumatoid arthritis (RA), affecting an estimated 2.1 million Americans, has been connected to interleukin-1 (IL-1), a cellular substance thought to cause the painful inflammation and debilitating erosion in rheumatoid joints. After January 1996 FDA approval, researchers injected cells genetically engineered to block the action of IL-1 directly into selected RA patients' joints. This gene encodes the interleukin-1 receptor antagonist protein, or IL-1Ra.
Seven of the nine gene transfer procedures approved by the FDA for this protocol have been completed, with preliminary, but promising results. No adverse effects have been reported. Gene expression has occurred in all patients tested thus far in the clinical trial, which provides a foundation for similar treatments of RA and related joint disorders.
"We are very encouraged by these results and are eager to begin the next phase
of the study," commented principal investigator Chris Evans, Ph.D., Henry J.
Mankin Professor of Orthopaedic Surgery at UPMC and director of UPMC's Ferguson
Laboratory for Orthopaedic
Research. "Now that we have established that the IL-1Ra gene is expressed, we
hope that the next trial will involve intervening at earlier stages of the
disease, with the intent of eliminating o
Contact: Lauren Ward
University of Pittsburgh Medical Center