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Arthritis and Gene Therapy As the "Baby Boomer" population grows older, more and more attention is being focused on conditions and issues associated with the aging process. Of particular interest are mobility-limiting disorders and conditions, such as rheumatoid arthritis. While the disease is, thus far, incurable and certainly hard to provide relief from, medical science has begun to focus on gene therapy as a potential source of limiting the effects of rheumatoid arthritis. Gene Therapy In a first of its kind gene therapy study beginning in the late 1990s, Drs. Christopher Evans, Paul Robbins, and James Herndon, explored the rheumatoid arthritis treatment possibilities offered by this brave new world that blends advanced bio-scientific technology with medical expertise. Evans' team noted at the outset that a number of anti-arthritic agents, such as the interleukin-1 receptor antagonist protein (IL-1Ra), show significant potential in the intervention of symptoms. The difficulty, however, was to develop a method of delivery. Since interleukin antagonists are proteins, it's not possible to administer them in the form of traditional medications—the digestive process would destroy them if administered in pill form, and they would break down quickly in the blood meaning to be effective large quantities would need to be administered. Something a little more radical was required. Options In looking at the various treatment delivery options, Evans and his associates felt that the use of gene therapy, i.e. literally using the body's own genes as a delivery system for the interleukins, may be effective. The researchers noted that by targeting the gene to the affected joint, much more of the agent reached the intended joint with far fewer side affects to the body as a whole. Evans and his team found that the most effective means of delivering the interleukin antagonists into the cells was to use viruses as delivery vectors. The viruses were modified so they were unable to replicate themselves and cause disease. These viruses were then exposed to cells extracted from the patient's finger joints, which they "infected" with the interleukin antagonist. The "infected" cells were then injected into the affected joints. A week later the joints were harvested and replaced with artificial joints. (An already scheduled joint replacement surgery was one of the requirements for recruitment.) The researches then evaluated tissue from the joints for changes. Results from this phase 1 clinical trial show promise. As more clinical trials are undertaken to explore this approach, new hope for rheumatoid arthritis patients increases. How ironic it would be if viruses, long thought of as a source of illness became a vehicle for cures! Resources Baum, M. (1996, July 18). World's first gene therapy for arthritis initiated at the University of Pittsburgh Medical Center. Retrieved August 5, 2002, from http://www.upmc.edu/NewsBureau/science/arthrel.htm. Skelly, A. (1999, March 2). Orthopedics Update: Gene therapy shows promise in nonfatal disease. The Medical Post, Vol. 35, No. 09. Retrieved August 5, 2002, from http://www.medicalpost.com/mdlink/english/members/ medpost/data/3509/20C.HTM.

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