A potential new Isis Pharmaceuticals ($ISIS) "anti-sense" drug helped restore normal muscle function in mice suffering from a form of muscular dystrophy. Credit new research spearheaded by Genzyme and the University of Rochester Medical Center with achieving the breakthrough.
The treatment targeted myotonic muscular dystrophy type 1, both the most common form of the disease and one of the two most severe iterations of adult-onset muscular dystrophy, according to National Institutes of Health statistics cited by the North Country Times story detailing the research news. And while mouse studies are an early step in a long drug development process with no guarantees, Isis has a lot to gain with moving forward. As the story reports, Isis recently inked a deal with Biogen Idec ($BIIB) that could be worth more than $270 million if it brings a viable muscular dystrophy drug to market, the story notes. Details are published in the journal Nature.
And there is progress here worth noting, despite the early stage of things. An adjoining Nature article points out, as the North Country Times notes, that the research at least maps out a framework from which to proceed, despite the amount of research that will be needed before humans can even test the drug.
How the chemicals work: They essentially stopped production of disease-causing proteins, working through Isis' gene-blocking anti-sense technology. Researchers have designed them to put out of commission defective RNA that messes up protein machinery in the body and can lead to a loss of muscle control and muscle weakness, cataracts and other problems. Normal muscle function follows the treatment, or at least it did in mice that were part of the trial. Researchers gave the anti-sense drug twice a week, over a month, to mice genetically engineered to show symptoms of the disease. The symptoms diminished for up to a year.
- read the North Country Times coverage
- check out the Nature study abstract
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