dichloroacetate

Dichloroacetate, commonly referred to as DCA, is the salt or ester of dichloroacetic acid, a small halogenated carboxylic acid. The chemical formula is C2H2Cl2O2 and the compound exists primarily as the dichloroacetate anion in solution. DCA is typically encountered as sodium dichloroacetate and is used in basic research; it is not approved as a pharmaceutical drug.

In biological systems, DCA inhibits pyruvate dehydrogenase kinase (PDK), reducing phosphorylation of the pyruvate dehydrogenase complex (PDH). This activation of PDH promotes the conversion of pyruvate to acetyl-CoA, increasing flux through the mitochondrial oxidative phosphorylation pathway and potentially altering cellular energy metabolism. The resulting metabolic shift has drawn interest for diseases characterized by mitochondrial dysfunction or cancer-associated metabolic reprogramming.

Clinical research: Since the late 1990s, DCA has been studied as a potential treatment for cancer and inherited mitochondrial disorders. Some early studies reported encouraging results, but subsequent larger or more rigorous trials have not established clear efficacy, and DCA is not approved by major regulatory agencies for cancer or other diseases. DCA is typically studied under medical supervision, often at off-label use, with dosing and duration varying across studies.

Safety and regulation: Reported adverse effects include peripheral neuropathy, reversible neurological symptoms, and liver enzyme elevations; there are concerns about toxicity and quality control, especially in non-clinical settings or dietary supplement products. DCA can interact with other medications, and long-term safety remains uncertain.

Chemistry and availability: DCA can be produced and sold for research purposes; it is not approved as a therapeutic agent. Handling requires standard chemical safety practices; it can be supplied as sodium dichloroacetate or dichloroacetate acid salts.