Edison

In the narrowest definition of inherited mitochondrial disease, there are an estimated 200,000 individual worldwide who carry this diagnosis. If re-defined to include diseases such as Parkinson’s, Huntington’s disease and stroke, the incidence exceeds millions. Despite this need, there is a remarkable absence of drugs to treat bona fide inherited mitochondrial diseases, or others with mitochondrial components.

Two strategies are being undertaken to develop treatments for these diseases. The first is a reductionist approach whose cornerstone is the development of a drug through a rationale understanding of the disease mechanism. The second is a more-so empiric approach, starting with the observation of partial clinical response to an existing drug, and improving its action absent an explicit understanding of disease mechanism. While there are pros and cons of both approaches, one significant observation has been made. Diseases of the mitochondria are responsive to one particular class of drugs– those based on the molecule co-enzyme Q10 (CoQ10).