Grant: Project Grant

Coenzyme Q10 (also known as CoQ10 or ubiquinone) is needed for mitochondria to produce energy from nutrients, as well as for other aspects of cellular functioning. It is also an well-known antioxidant that protects from free radical damage. There are patients whose primary problem is that they produce insufficient amounts of CoQ10. This leads to severe symptoms that resemble the symptoms produced when mitochondria do not function well. In addition, there are a number of other diseases that lead to lower levels of CoQ10 in cells, which has deleterious consequences. Aging also lowers the levels of CoQ10 in the body. Currently, the treatment for all these situations is to give oral CoQ10 as a food supplement. Unfortunately, very little CoQ10 enters the body by this method. In this application, we propose to develop and study two new methods that we have discovered to increase CoQ10 in the body. One method puts CoQ10 in a state in which it can be given by intravenous injection, something that was not possible until now, and that we have shown to be able to greatly increase the amount that can be delivered into the body compared to what can be done by giving it as food supplement. Now we want to show that administering CoQ10 in this manner indeed relieves the deleterious consequence of low CoQ10 levels. The other method is based on our discovery of a drug that is not toxic and already approved as therapy for another diseases. We found that this drug increases CoQ10 in the body by slowing down the rate at which CoQ is normally lost from cells and from the body. Thus, it can boost the amount of CoQ10 in cells, even in patients that cannot make normal amounts of CoQ10.