April 23, 2008

High-Dose Coenzyme Q10
Shows No Benefit in ALS

Principal investigator Petra Kaufmann at Columbia University Medical Center in New York, with colleagues at 19 U.S. centers, have found that high-dose coenzyme Q10 (coQ10) is not promising enough to warrant further study as a treatment for ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

The findings were announced April 16 at the 60th annual meeting of the American Academy of Neurology in Chicago. The study was funded by the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health in Bethesda, Md.

The investigators tested coQ10 at dosages up to 2,700 milligrams per day against an inactive substance (placebo) in 185 people with ALS. Coenzyme Q10 was considered a promising treatment for ALS because it acts in the mitochondria, the “powerhouses” of the cell, and is also a powerful antioxidant, Kaufmann said.

“The difference between the 2,700-milligram coQ10 group and the placebo group was not large enough to suggest that one would find a meaningful difference in a future phase 3 [large-scale] trial,” Kaufmann said. She added, however, that the study didn’t address the question of whether coQ10 might be helpful in combination with other drugs.

Kaufmann said the result was “disappointing because we didn’t find a drug that helps people with ALS.” However, she noted, it frees ALS patients from believing they have to buy coQ10, and it frees resources to test other drugs rather than conducting an expensive, phase 3 trial of coQ10 in ALS.

Kaufmann thanked NINDS and the investigators, but said, “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. Trials allow us to rationally test treatments, eliminate harmful or ineffective treatments rapidly and, we hope, find an effective and safe treatment for ALS soon.