Mitochondrial Medicine

The Mitochondria and Cancer Connections (MC²) Research program leverages knowledge from existing centers to establish a novel, cutting-edge research initiative and enable the development of mechanistic understanding and novel therapeutic opportunities at the intersection of mitochondrial disease and pediatric cancer.

We are recruiting patients with genetically confirmed primary mitochondrial myopathy who are at least 18 years old for a randomized, double-blind, placebo-controlled study of a new drug to treat this disorder.

We are studying an investigational drug called sodium dichloroacetate (DCA).

The Mitochondrial Medicine Research Laboratory investigates the genetic etiologies, pathophysiologic mechanisms, and therapeutic targets of mitochondrial disease.  The lab applies state-of-the-art technologies and approaches in diverse animals (C. elegans worms, D. rerio zebrafish, M.