AskBio Launches Human Gene Therapy Trial in Battle Against Muscular Dystrophy

is launching the first human gene therapy trial targeting Duchenne muscular dystrophy.

Asklepios, which is better known as AskBio, is working with the Columbus Children's Hospital in Ohio and the Muscular Dystrophy Association (MDA) in the trial.

In 2004, the MDA awarded AskBio a $1.6 million grant, the largest in its history, to help underwrite the cost of the Chapel Hill-based firm's research.

Andrew Kilbarger, an 8-year-old from Lancaster OH, received an injection of a drug called Biostrophin that is being developed by AskBio.

The Food and Drug Administration granted approval for the trial on March 3. In all, six boys will participate in the trial with each receiving a Biostrophin injection in one arm and a placebo in the other.

"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," said neurologist Jerry Mendell, who injected the drug. "We are all hopeful that this effort will help guide us toward clinically meaningful results as we move forward with this form of treatment."

Mendell is co-director of the MDA clinic at the Columbus hospital. He also is head of the Neuromuscular Research Program and Gene Therapy Center at Columbus Children's Research Institute and is a professor of pediatrics, neurology and pathology at Ohio State.

DMD occurs when a gene on the X chromosome fails to make the essential muscle protein dystrophin. One of the purposes of the trial is to see if the drug injection leads to production of dystrophin.

AskBio has developed a means of delivering gene-based drugs through the development of biological nano particles (BNPs). Duchene's muscular dystrophy was among its early targets. AskBio was launched in 2002.

"Gene therapy is expected to provide therapeutics for certain types of diseases that have not been able to be successfully treated by conventional medicine," Sheila Mikhail, AskBio's chief executive officer, told WRAL Local Tech Wire in a 2005 interview. "However, gene therapy has not become a realistic means to treat patients because the mechanisms to deliver genes have exhibited shortcomings."

The launch of the trial is the latest benchmark to be reached in a 20-year research endeavor, said Sharon Hesterlee, director of research development for the MDA. She noted that the gene for dystrophin was discovered in 1986.

"In cases like this, we've had to move beyond academic institutions, where basic science concepts are developed, and into industry, where concepts can become products," Hesterlee said. "Asklepios' willingness to take a big risk allowed us to build this unique coalition of business, academic and nonprofit players. This is a true collaborative effort."

Professors Xiao Xiao of the University of Pittsburgh and Jude Samulski of the University of North Carolina at Chapel Hill have worked on Biostrophin for 20 years. They are the co-founders of AskBio.

"After years of encouraging preclinical results, I'm excited that AskBio will soon be able to bring this promising new therapy into the clinic, and look forward with a great deal of optimism to offering this initial step toward hope for the DMD community," Samulski said in a statement.