Amyotrophic Lateral Sclerosis - Clinical Research

Clinical Research

A number of clinical trials are underway globally for ALS; a comprehensive listing of trials in the US can be found at

Thalidomide and Lenalidomide have shown efficacy in protecting motor neurons in transgenic (G93A) mice.

KNS-760704 (Dexpramipexole) is under clinical investigation in ALS patients. It is hoped that the drug will have a neuroprotective effect. It is one enantiomer of pramipexole, which is approved for the treatment of Parkinson's disease and restless legs syndrome. The single-enantiomer preparation is essentially inactive at dopamine receptors, is not dose limited by the potent dopaminergic properties of pramipexole. Results of a Phase II clinical trial conducted by Knopp Neurosciences and involving 102 patients were reported in 2010; the trial found a dose-dependent slowing in loss of function. A larger phase II trial conducted by Biogen found the drug to be safe, well tolerated, and associated with a dose-dependent slowing in the decline of ALS.

A Phase II trial on talampanel was completed by Teva Pharmaceutical Industries in April 2010 however it was found to be negative for treatment viability.

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