Dr. Jerry Mendell, chairman of the Department of Neurology at the Ohio State University Medical Center in Columbus, injected a muscle on the top of Decker's foot with genes for a muscle protein that's missing because of a genetic flaw. The gene therapy trial was authorized by the Food and Drug Administration, and received a recommendation from the National Institutes of Health Recombinant DNA Advisory Committee earlier today. We're making final preparations for the Telethon this weekend, and we've now set the stage for what very well could be the payoff we've all been working so hard to achieve. Gene therapy is the insertion of a working gene to compensate for genetic flaws, such as those that lead to muscular dystrophy, a progressive muscle-wasting disease that is debilitating and often fatal. LGMD, a form that affects tens of thousands of Americans, mostly damages the muscles that stabilize the hips and shoulders, is generally slowly progressive and often leads to wheelchair use by middle age.
Serious complications can result if the heart and respiratory muscle become involved.
So seeing gene therapy go from the rigors of the laboratory to the first human trial for muscular dystrophy is especially meaningful. Stedman was present for the historic injection, which took place at OSU Medical Center. MDA grantee Kevin Campbell, a professor in the Department of Physiology and Biophysics at the University of Iowa, has identified several key proteins involved in different forms of muscular dystrophy. He determined the eligibility of patients scheduled to participate in this trial on the basis of molecular analysis of their muscle cells. It's kind of been a long waiting period, but this is the first step of the cure.