New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders—complex conditions with severe energy deficiency for which no proven effective treatments exist. Future clinical research is needed to explore whether the drug, cysteamine bitartrate, will meaningfully benefit patients.
Certain defects in the way the mitochondria work may lead to diabetes, and the ways our bodies adapt to high blood sugars share many features with the ways our bodies respond to mitochondrial disease.
