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Release: Sept. 7, 1999

UI research makes possible gene therapy trial for MD

IOWA CITY, Iowa -- The efforts of a University of Iowa Health Care researcher and members of his lab are making it possible for researchers at another university to begin testing what could be a promising treatment for limb girdle muscular dystrophy: gene therapy.

Kevin Campbell, Ph.D., UI professor of physiology and biophysics, and neurology, and a Howard Hughes Medical Institute Investigator, has spent much of his career focused on understanding and trying to find ways to treat muscular dystrophy, which is a group of hereditary diseases characterized by progressive muscle weakness and degeneration.

On Thursday, Sept. 2, Ohio State University researchers started the first human study looking at gene therapy as a way to treat limb girdle muscular dystrophy, thanks in part to Campbell's work. The limb girdle type of muscular dystrophy involves initial deterioration of shoulder and pelvic girdle muscles, with relative sparing of other muscle groups. It is thought that limb girdle muscular dystrophy affects between one in 20,000 and one in 50,000 individuals.

Many researchers believe that gene therapy is one of the most promising possibilities for treating muscular dystrophy, as well as many other genetic diseases. Gene therapy involves using a vector, often a disabled cold virus, as a vehicle to supply cells with the needed materials to correct a defect and thus treat a disease and/or its symptoms. In the case of limb girdle muscular dystrophy, researchers injected the muscles' cells with an adenovirus carrying a protein particle called alpha sarcoglycan, which is necessary to protect a muscle from becoming damaged.

Campbell and members of his lab have been instrumental in many important, early advances in muscular dystrophy research. Campbell and his team were the first researchers to explain the basic science of how the defective sarcoglycan complex causes limb girdle muscular dystrophy.

The Campbell lab was also the first group to demonstrate functional restoration of a muscle using gene transfer and to develop an animal model on which to effectively test the gene therapy. In June, at the American Society of Human Gene Therapy meeting in Washington, D. C., the Campbell

group presented findings that showed how the gene therapy technique causes large regions of muscle to express the corrected defect.

"This work has now made it possible to begin human trials," Campbell said. "The work at Ohio State is an initial study, but it is an exciting start. The goal of this initial trial is to make sure that the treatment is safe and to find out how the immune system responds to the gene therapy."

Ohio State researchers have begun testing the approach on a small muscle in the foot. The trial involves six patients, one of whom was an individual initially diagnosed by the Campbell lab.

"If initial studies are safe, then the investigators will start to inject larger muscles," said Campbell, who will help analyze the muscle tissue biopsies.

A second trial would involve multiple medical centers. The UI would likely participate in the expanded investigation, which would be designed to test the effectiveness of the gene therapy.

While the gene therapy strategy is promising, Campbell indicated that there are still many issues to consider. He also cautioned against thinking of gene therapy as a possible cure for muscular dystrophy at this time.