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New drug may boost muscle strength in people with ALS

Thursday 01 March 2012

Researchers at biotech firm Cytokinetics have developed a drug called CK-357 that can potentially boost muscle strength in people with Amyotrophic Lateral Sclerosis (ALS). CK-357 sensitizes muscle cells to the calcium that activates contraction, making them flex even if the incoming signals from neurons are weak. Cytokinetics is currently testing the drug in Phase 2 trials for ALS and myasthenia gravis, an autoimmune neuromuscular disease.

In the first phase of the trial, researchers provided the drug once a day over a two-week period to 24 people with ALS. CK-357 was well tolerated, and a side effect of dizziness tended to wear off during the trial period. While the study was underpowered to detect clinical benefits, some people seemed to improve in their ability to perform daily functions and in their breathing ability. Now, Cytokinetics is testing CK-357’s effects when given concurrently with the ALS drug riluzole in another 24 people.

"If you can make the muscles a little bit stronger, it would make a big difference for people with ALS," said Dr. Robert Miller, director of the Forbes Norris MDA/ALS Research Center, who was not involved in the study. The drug boosts strength by 15-25 percent in healthy people, noted Miller. For a person with ALS, that kind of increase could mean the ability to get around as well as to eat, bathe, and dress independently, he said.

http://www.nature.com/nm/journal/v18/n3/full/nm.2618.html