Current treatments for mitochondrial disease focus mainly on treating symptoms and slowing down the disease’s progression. Common treatments include dietary therapy, vitamin supplements, and lifestyle changes to reduce physiological stress (such as that produced by extremes of heat and cold).
As with many other illnesses, stem cells may provide new options for treating many types of mitochondrial diseases. However, because each of the more than forty types of mitochondrial disease have different causes, it is not likely that one type of therapy will work effectively for all types of mitochondrial disease.
There is hope, however, for treatment of certain types of mitochondrial disease. At a meeting on mitochondrial diseases held in Phoenix, AZ in May 2008, researcher Michio Hirano of Columbia University in New York spoke of a possible treatment for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), a type of mitochondrial disease caused by a genetic defect in a nuclear enzyme which is responsible for breaking down thymidine. The failure of the enzyme causes thymidine to accumulate in the blood, at levels which are toxic to mitochondria.
The experimental treatment for MNGIE developed by Dr. Hirano involves infusions of healthy blood stem cells, intended to restore function of the required enzyme, and eliminate thymidine toxicity. At the May 2008 meeting, Hirano reported that one patient, a thirty year old woman with MNGIE, had experienced a remarkable reduction in symptoms up to two and a half years after receiving donor blood stem cell infusions.
