Dystrophin Gene Transfer safe in Duchenne muscular dystrophy
An Muscular Dystrophy Association supported trial to transfer genes for the muscle protein dystrophin, needed but missing in Duchenne muscular dystrophy (DMD), has shown that the procedure is safe and well tolerated in six boys. Researchers plan to test three additional patients at a higher dosage level.

In this trial, which began in March 2006, researchers injected a dystrophin gene compound called Biostrophin, which was developed with MDA support at Asklepios Biopharmaceutical in Chapel Hill, N.C., into the biceps muscles of boys with DMD.
Biostrophin is a combination of miniaturized dystrophin genes and adeno-associated viral shells that home to muscle fibers.

“The patients have been injected with the gene carried by an adeno-associated virus into one muscle of the arm,” said neurologist Jerry Mendell, director of the Gene Therapy Center and the clinician on the study. He also serves as co-director of the MDA clinics at Nationwide Children’s and Ohio State University Hospitals in Columbus. Mendell injected the children at Nationwide Children’s Hospital.

“The patients have been carefully followed for side effects of the treatment, and none have been encountered,” he said. “This is primarily a safety trial, and we can confidently report that safety has been achieved. An additional goal is to lay the ground work for future gene therapy trials by establishing the ideal dose for treatment. In this trial, two doses have been tested, and another will be required before completion of the study.” Mendell said the three additional trial participants will “receive a higher dose, which by all indications will be safe to administer.” {No participants are being recruited at this time.}

“Upon completion of the trial in nine subjects, we will be able to report to the scientific community and the public the results of the trial,” he said, “with recommendations for future gene therapy trials for this devastating form of muscular dystrophy.”