CLINICAL TRIAL: 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 a devastating disease of the nervous system. It leads to progressive loss of those nerve cells that control muscle bulk and movement. Patients develop increasing weakness in all muscles, including those needed for breathing, swallowing and speaking. ALS typically leads to death within 3 to 5 years of onset. Research results implicate that mitochondria, the powerhouse of the cell, are affected in ALS. Other data suggests that "free radicals", very reactive oxygen species, contribute to the nerve cell damage. Coenzyme Q10 (CoQ10), a natural nutrient, helps mitochondrial function and scavenges free radicals. It has prolonged survival in a mouse model of ALS and it has helped patients with Parkinson's disease, another disease caused by nerve cell loss. In two small studies we have given CoQ10 to patients with ALS and found that it was well absorbed by the body and also safe and well tolerated. Therefore, CoQ10 is a promising treatment for ALS. We propose a clinical trial with CoQ10 for patients with ALS. The trial will have two stages. In a first stage we will determine the best of 2 doses. We have selected the two doses based on the results of our smaller studies. In a second stage we will test whether CoQ10 is more effective than placebo, a sugar pill without any active ingredient. The treatment period for each patient will be 9 months. All participants will be allowed to take riluzole, the only currently approved treatment for ALS.