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.
Energy continues to build for the role of the mitochondrion in health and disease, a field pioneered by Douglas Wallace, PhD, director of the Center for Mitochondrial and Epigenomic Medicine at Children's Hospital of Philadelphia.
What causes disease? Why do we get sick? Douglas Wallace's answer could spark a revolution in medical thinking. For thousands of years, practitioners of Eastern medicine have linked human health to the concept of energy, or "qi" - its presence, absence and flow through our bodies affecting everything from heart disease to mental clarity. Ironically, Douglas Wallace, PhD, one of CHOP's most internationally renowned experts in Western medicine, has spent his career proving that they just might be right.