Disease reversed in mice

It’s a breakthrough that has made top Toronto muscular dystrophy researcher Christopher Pearson almost giddy with hope.

It’s a breakthrough that has made top Toronto muscular dystrophy researcher Christopher Pearson almost giddy with hope.

“There’s light at the end of the tunnel, which freaks me out,” the senior Hospital for Sick Children scientist says of research that shows a new way to attack myotonic dystrophy, the ailment’s most common form, at its genetic root.

“I’ve been (working on) this for over 10 years and I didn’t think I’d see it in my lifetime. But it’s happening.”

What’s “happening” is contained in a University of Rochester study — headed by neurologist Dr. Charles Thornton— published yesterday in the journal Science, suggesting the wasting, muscle-locking effects of myotonic dystrophy can be reversed by combating it at the DNA level.

Myotonic dystrophy attacks one in 7,500 people in North America, and as many as one in 400 across regions of Quebec.

Symptoms include involuntary tightening of the hands and limbs and progressive muscle wasting that can erupt in childhood and span decades.

Thornton’s team injected a potential new miracle molecule into the muscles of mice genetically manipulated to mimic the human disease.

The rodent muscles not only ceased their deterioration, but actually returned to normal function, Thornton says.

The research has sparked new hope for Toronto’s Robert Harrison, 48, whose body has become more and more immobilized since he was diagnosed 13 years ago.

“Any breakthrough gives me hope that it can help me out,” says Harrison.

Thornton says the major challenge now is to devise a delivery system that can bring the molecule to afflicted muscle, heart and central nervous system tissues.

 
 
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