CoQ10 Trial Results

From the Desk of Dr. Kaufmann

CoQ10 is not promising enough to warrant further study as a treatment for ALS – Early Results from the "Clinical Trial of High Dose Coenzyme Q10 (CoQ10) in ALS - QALS"

Thanks to the participation of 185 people with ALS and the contributions of nineteen ALS research teams across the United States, we are now able to report the results of QALS. The trial was a successful collaborative effort between researchers, clinicians and people with ALS in that we met our recruitment targets and completed the trial in a timely fashion.

CoQ10 is a promising treatment for ALS because it is a mitochondrial co-factor and powerful antioxidant. Mitochondria are the "powerhouses" of the cell, and they are thought to have impaired function in ALS. Free radicals that can be neutralized with antioxidants are also thought to play a role in ALS.

We therefore conducted a Phase II clinical trial, funded by the National Institute for Neurological Disorders and Stroke. We wanted to find out if it is worthwhile pursuing CoQ10 as a treatment for ALS in Phase III. Our trial was not designed to definitely answer the question if CoQ10 is actually beneficial in ALS. Only a Phase III trial can show this. You may ask why we did not start a Phase III trial right away. The reason is that Phase III trials are very large, expensive and take a long time. When there are several new drugs on the horizon, it is an efficient strategy to test some of them in Phase II so that we can have at least preliminary answers more quickly and without using up all the resources.

The results for our primary outcome suggest that CoQ10 at 2700 mg daily is not promising enough to warrant further studies as a treatment for ALS. The difference between the CoQ10 group (2700mg daily) and the placebo group was not large enough to suggest that one would find a meaningful difference in a future Phase III trial. Our study did not address the question of whether CoQ10 may be promising in combination with other approved or experimental drugs, but usually the first step in assessing combination therapies is to investigate the efficacy and safety of the individual compounds.

Although this result is disappointing because we did not find a drug that helps people with ALS, it is in a way positive that we were able to obtain this information. The reason is that many people with ALS think that they have to buy CoQ10 and that it will help them. Now, we know this may not be worthwhile based on our results showing that it is unlikely that there is more than a small effect. Also, we think that our study helps the ALS community. This is because we conclude that larger studies with CoQ10 at 2700mg daily are not warranted. Therefore, other drugs that are more promising can be tested as no further resources will have to be used to test CoQ10 in ALS.

We want to thank NINDS for funding, and the investigators for their support and their contributions to this trial. Most of all, we want to thank people with ALS and their caregivers who have participated and generously given their time and effort. Clinical trials can not only benefit those who directly participate, but they can help others with ALS. Clinical trials allow us to rationally test treatments, eliminate harmful or ineffective treatments rapidly, and, hopefully, find an effective and safe treatment for ALS soon.