Saturday, October 20, 2007

Gene Therapy Trial for DMD

Duchenne muscular dystrophy is an inherited degenerative disease that affects voluntary muscles. Approximately 1 in every 3500 boys born have this disease. And all ethnic groups are equally susceptible to DMD. You can learn more about DMD from the "Your Genes Your Health" site, as well as the MDA website and the Muscular Dystrophy Canada site. Here are a few details about the disease from the MDA site:

Duchenne muscular dystrophy

Cause - An absence of dystrophin, a protein that helps keep muscle cells intact.

Onset - Early childhood - about 2 to 6 years.

Symptoms - Generalized weakness and muscle wasting first affecting the muscles of the hips, pelvic area, thighs and shoulders. Calves are often enlarged.

Progression - DMD eventually affects all voluntary muscles, and the heart and breathing muscles. Survival is rare beyond the early 30s.

Today's "Google Alerts" brings this encouraging story about the world's first clinical trial for gene therapy for DMD. Here are a few excerpts from the story:

A gene therapy trial for the fatal disorder Duchenne muscular dystrophy (DMD) is about to begin in London.

In a world first, a small group of patients will be injected with an experimental drug which it is hoped will extend their lives.

....The injection contains a "molecular patch" targeting the faulty gene so that it should work again.

,,,,Professor Muntoni describes the gene therapy as like a piece of molecular velcro which will form a temporary repair.

Gene therapy will work best in young children, as it cannot reverse existing muscle damage.

....The Muscular Dystrophy Campaign said it had high hopes the outcome of the study would be positive.

Dr Marita Pohlschmidt, the campaign's director of research, said: "The start of the trial is exciting news because it will provide us with the evidence of how this promising technology will prevent the wasting of muscles in boys with Duchenne muscular dystrophy.

"This is the first time that results from this type of research are so close to being translated into a treatment.

"We are proud that we were able to play a vital role in the formation of the scientific consortium and to support the preclinical work with more than £500,000."