CLINICAL TRIAL OF HIGH DOSE COQ10 IN ALS

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Amyotrophic lateral sclerosis (ALS) is one of the most devastating neurological diseases leading to death typically in 3 years of onset. Treatment is severely limited, and no cure is available. Oxidative stress and mitochondrial dysfunction have been implicated in ALS pathophysiology. Coenzyme Q10 (CoQ10), a mitochondrial cofactor and powerful antioxidant, prolongs survival in the transgenic ALS mouse model and slows functional decline in other human neurodegenerative disease. CoQ10 is a nonprescription dietary supplement with an excellent safety profile and central nervous system penetration. Thus, we propose to conduct a 2-stage, phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial of high dose CoQ10 against placebo (target enrollment 185 patients at 18-20 clinical sites).