Band-Aid for Muscular Dystrophy

June 21, 2009 5:18:00 PM PDT
Muscular dystrophy is one of the most common genetic disorders. One in every 3,500 baby boys is diagnosed with the condition. The most devastating type, Duchenne muscular dystrophy, slowly robs kids of their muscles, and they rarely live past age 20. Researchers said they've made a drug discovery that could give kids a fighting chance.

He's gradually getting weaker, but his star power is stronger than ever. Drew Bonner is an inspiration to kids with Duchenne muscular dystrophy, a fatal genetic disorder where muscles waste away.

"I said, OK, what are the treatments, where do we go from here?" Drew's mother, Jan Bonner, told Ivanhoe. "We can deal with this, and the doctor told us there are no treatments. There are no cures."

Drew doesn't depend on a wheelchair yet, but leans on others to get around. "It's a challenge I have to overcome," Drew told Ivanhoe. "It's one more hurdle to jump over."

Researchers say they've cleared a huge hurdle in finding a treatment. "It's at our fingertips, getting these kids out of their wheelchairs," Eric Hoffman, Ph.D., Professor of Pediatrics at Children's National Medical Center in Washington, D.C., told Ivanhoe.

Dr. Hoffman is part of a worldwide team developing the technique called exon skipping. The injectable drug cocktail works like a band-aid, covering up the mutation that causes muscle weakness and allowing cells to make a healthier protein that improves muscle function.

"It's almost if you were reading a recipe and instead of putting salt, your mutation said put in lots of hot pepper instead," Dr. Hoffman explained. "Well, your cake wouldn't be so great. This band-aid lets it skip over that wrong pepper instruction."

When tested in dogs with muscular dystrophy, the animals went from struggling to walk to running. The canine version of the disease affects the same gene as the human version.

"Time is not on our side," Jan said.

Drew understands the urgency, but refuses to be defined by a disease.

"How's life any fun if you're just gonna be a downer?" he said. "Why not just live it and be happy?"

He's a boy who's focused on being 13, while doctors work on finding his cure.

Researchers say this strategy could help up to 90 percent of people affected by Duchenne muscular dystrophy. Trials involving people are underway in Europe.

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