MUSCULAR DYSTROPHY GENE THERAPY TRIAL
PASSES FIRST TEST BY NIH COMMITTEE

TUCSON, Ariz., Dec. 16, 2004 — The Recombinant DNA Advisory Committee (RAC), part of the U.S. National Institutes of Health (NIH) in Bethesda, Md., today voted that the first American study of gene therapy in boys with Duchenne muscular dystrophy (DMD) can move forward, the Muscular Dystrophy Association (MDA) announced.

The study reviewed by the RAC will test the safety of a laboratory-engineered gene for the muscle protein dystrophin tucked inside a modified adeno-associated virus. Dystrophin is a protein needed but missing in boys and young men with DMD.

In the MDA-funded trial, six boys with DMD, a devastating childhood muscle disease that affects some 30,000 boys in the United States and results in death from respiratory or heart failure in the 20s, will undergo injections of the gene into their biceps muscles. One biceps will receive gene injections, while the other receives sham injections.

After about six weeks, samples of muscle cells from each biceps will be examined for evidence of dystrophin production and signs of any damaging reactions, such as an unwanted immune response.

The study participants, who will receive injections and monitoring at Columbus Children’s Research Institute, part of Ohio State University, will undergo many other types of safety testing during and after the gene transfer.

“A successful review from the RAC means that we have passed the first hurdle in initiating a gene therapy trial for DMD in the United States,” said Sharon Hesterlee, MDA director of Research Development. “The project is funded as a milestone-driven contract through our new Translational Research Program.”

The gene therapy trial will be conducted under the auspices of a Chapel Hill, N.C., biotechnology company, Asklepios Biopharmaceutical Inc., which includes researchers from Ohio State University, the University of Pittsburgh and the University of North Carolina. Asklepios has received a grant of $1.6 million from MDA for this trial. The grant, awarded in July, is the largest ever made by MDA to a private company.

A review by the RAC is mandatory for this type of gene therapy trial. The proposal still must be approved by the Food & Drug Administration, which is expected to review the project as early as next summer.

MDA is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. For more information about MDA, call (800) 572-1717 or visit www.mda.org.