Muscular dystrophy reversed in mice with Isis Pharmaceuticals research

Symptoms of a form of muscular dystrophyhave been reversed in mice, a research team including scientists from Isis Pharmaceuticals Inc.reported Wednesday.

Chemicals injected into the mice rapidly reduced levels of toxic molecules that cause the disease, the scientists said. They reported their findings in the scientific journal Nature.

It will take much more research before the treatment can be tried in humans, said an accompanying news article in Nature. "However, the path to success now seems clearly visible," the article stated.

The chemicals work through Isis' gene-blocking antisense technology, which stops production of disease-causing proteins. The technology represents Isis' bid to become a powerhouse in discovering new drugs. Antisense drugs can theoretically work on a vast number of diseases.

Shares of Isis, which is based in Carlsbad, closed Wednesday at $11.80, a loss of 32 cents for the day. That's still about 63 percent higher than Isis' close of $7.25 on Jan. 3, the first trading day of 2012.

The team included Frank Bennett, senior vice president of research at Isis, and two other Isis scientists. Others are scientists at the University of Rochester Medical Center andGenzyme.Charles Thornton, a University of Rochester neurologist, was senior author.

The disease is called myotonic muscular dystrophy type 1. Isis recentlysigned a dealwith Biogen Idec to find a drug for the disease. The deal could bring Isis more than $270 million if it discovers a drug that makes it to market.

The type 1 form of the disease is the commonest and most severe of two types of adult-onset muscular dystrophy,according tothe National Institutes of Health.

Myotonic dystrophy causes progressive muscle wasting and weakness, according to the NIH. Patients often experience prolonged muscle contractions, or myotonia, and can't relax certain muscles after use. Patients may also have slurred speech or temporary locking of the jaw.

Other symptoms include cataracts, abnormal electrical signals in the heart, and in men, early balding and infertility. In type 1 patients, muscle weakness especially affects the lower legs, hands, neck and face.

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Muscular dystrophy reversed in mice with Isis Pharmaceuticals research