See on Scoop.it – ALS Lou Gehrig’s Disease
There has never been much innovation to brag about in the treatment of the neurodegenerative disease that killed Yankees legend Lou Gehrig. But South San Francisco-based Cytokinetics is reporting today on clinical trial results that offer a hint it could be onto something for treating amyotrophic lateral sclerosis (ALS). An experimental drug from Cytokinetics met its main goal of demonstrating safety in 49 patients, while showing signs after two weeks of beating a placebo on a standard test of disease progression, according to results presented today at the American Academy of Neurology meeting in New Orleans. The result was from a small study which wasn’t designed to show a clinical benefit, and the findings were not statistically significant, meaning it could have been a fluke. But the finding is provocative enough that Cytokinetics has been emboldened to take the next step, and invest significant time and money to advance to the final phase of clinical trials normally required for FDA approval of a new drug. “The idea you can see a change after two weeks on a functional rating scale is encouraging,” says Jeremy Shefner, the chair of the Department of Neurology at the Upstate Medical University at the State University of New York, and the study’s principal investigator. “It’s something we’ve never seen in ALS before.”
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