Gene Therapy Trial for Duchenne Muscular Dystrophy Begins

[Angel]

t's the first study of its kind in the U.S.

The first U.S. gene therapy trial focused on Duchenne muscular dystrophy is under way at Columbus Children's Hospital in Columbus, Ohio.

"This is a long-awaited step in the overall plan to help kids with muscular dystrophy, especially for those of us who have been involved in clinical trials for many years," neurologist Dr. Jerry Mendell, co-director of the Muscular Dystrophy Association Clinic at Columbus Children's Hospital, said in a prepared statement. "We are all hopeful that this effort will help guide us toward clinically meaningful results as we move forward with this form of treatment."

The study will assess the safety and efficacy of a functional gene for the muscle protein dystrophin. Duchenne muscular dystrophy occurs when a gene on the X chromosome fails to make this essential muscle protein. The disease begins in early childhood and causes progressive loss of muscle strength and bulk. People with the disease usually die in their 20s from respiratory or cardiac muscle failure.

Six Duchenne-affected boys will take part in the study. Each boy will receive an injection of dystrophin genes in the muscle of one arm and a placebo in the other arm. Neither the boys nor the researchers will know which arm received the genes.

After several weeks, the researchers will analyze samples of muscle tissue from both arms of all the study participants, who will also undergo strength and health testing. The researchers will assess whether this gene therapy is likely to result in persistent production of dystrophin in muscle cells.