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August 29, 1996

A first: gene therapy used to treat patient with rheumatoid arthritis

University of Pittsburgh Medical Center (UPMC) doctors on July 17 performed the world's first gene therapy procedure on a patient suffering from rheumatoid arthritis.

The trial marks a transition in the use of gene therapy from patients with fatal diseases, such as cancer or AIDS, to disabling disorders like arthritis.

The trial also is the first attempt to use gene therapy on both an autoimmune disorder, one in which a person's immune system attacks the body's own tissue, and on connective tissues.

In the procedure, gene therapy is used to block cellular changes that lead to erosion and inflammation in joints affected by rheumatoid arthritis.

"This gene therapy is unique," said Chris Evans, principal investigator. "It opens a new chapter for the treatment of arthritis, other autoimmune diseases and additional disorders of bones and joints." Doctors involved in the study are hopeful that gene therapy can someday be used to stop the biological processes underlying disorders such as arthritis.

"Current therapies reduce pain and inflammation, but they do not stop the progress of rheumatoid arthritis, which this study is designed to address," said James Herndon, chair of UPMC's Department of Orthopaedic Surgery and the surgeon who performed the operation.

An estimated 40 million Americans suffer from some form of arthritis. The disease costs the economy about $64.8 billion annually in medical care and indirect costs such as lost wages.

As baby boomers age, the prevalence of arthritis is expected to increase 57 percent by the year 2020, according to the Arthritis Foundation. Rheumatoid arthritis alone afflicts 2.1 million Americans, mostly women.

The 68-year-old woman who was treated in the trial received injections of her own cells into the knuckles of one hand. These cells were cultured and modified to carry a gene that blocks both joint erosion and inflammation caused by interleukin-1 (IL-1), a biological response modifier.

Normally, IL-1 stimulates inflammation as part of the body's immune system to ward off infection or disease. In rheumatoid arthritis, IL-1 inflammation turns and attacks a person's joints, causing inflammation and erosion of joint tissues, as well as limited mobility and chronic pain.

Preliminary results from this study are not expected until late fall. Early stages of the trials will involve a total of nine women with rheumatoid arthritis who already are scheduled to receive joint replacements.

The gene therapy trials are being supported by UPMC's Department of Orthopaedic Surgery, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute for Digestive Disorders, Diabetes and Kidney Diseases, and Orthogen GmbH, a German pharmaceutical company.

Pitt already has filed various patent applications related to the new technology.

UPMC researchers have additional preliminary data from animal models that suggest this form of gene therapy may be beneficial for patients with osteoarthritis. The most common form of arthritis, osteoarthritis erodes joints due to aging, wear and tear, and injuries.

Filed under: Feature,Volume 29 Issue 1

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