Study Shows Rheumatoid Arthritis Responding To Gene Therapy
A study that delivers the first clinical evidence that gene therapy can reduce the symptoms of patients with rheumatoid arthritis appeared in the February issue of Human Gene Therapy. The authors of the study, which was carried out in 1997 and 1998 under the direction of Dr. Peter Wehling, Düsseldorf, Germany, describe the findings of a study involving two patients with severe rheumatoid arthritis.
In gene therapy, genes inside the cells are modified so as to either repair a genetic defect or to trigger the cells to produce a protein that stops the disease. The first clinical studies of such techniques began in 1990 for the treatment of rare genetic defects of the immune system. The gene transfer technique used in the present study was approved by the Ethics Committee of the Düsseldorf University Hospital.
Rheumatoid arthritis is a classic auto-immune disorder in which the body’s immune system turns against itself, resulting in swelling and inflammation of the joints. The disease causes permanent destruction of joint tissue, especially cartilage, and remains incurable. It is estimated that 5-10 million patients of the EU suffer from rheumatoid arthritis. “Rheumatoid arthritis is an extremely painful condition that can affect multiple joints of the body. It is an ideal indication for this technique because the joint forms a closed space. The genetically modified cells can simply be injected into the joint because they will stay in place”, notes Dr. Rüdiger Krauspe, director of the Orthopaedics Clinic at Düsseldorf University Hospital.
Lead investigator Peter Wehling explains the technique: “We injected genetically modified cells taken from the patient’s own body into the diseased joint. This stimulated the production of the human interleukin-1 receptor antagonist, which stops the breakdown of cartilage by blocking the interleukin-1 protein that is responsible for the inflammatory processes.“ “Essentially the gene becomes its own little factory, continuously working to alleviate pain in the joint “, says Christopher H. Evans of the Center for Molecular Orthopaedics, Harvard Medical School, Boston, one of the co‑authors of the study.