Dichloroacetate by Ray Sahelian, M.D.
Dichloroacetate (DCA) is a potential environmental hazard, owing to its wide presence in the biosphere and its association with animal and human toxicity.
Dichloracetate benefit
Therapeutic potential of dichloroacetate for pyruvate dehydrogenase complex
deficiency.
Mitochondrion. 2006 Jun;6(3):126-35. Division of Endocrinology and
Metabolism, Department of Medicine, University of Florida College of Medicine,
P.O. Box 10226, Gainesville, FL 32610, USA.
We reviewed the use of oral dichloroacetate in the treatment of children with
congenital lactic acidosis caused by mutations in the pyruvate dehydrogenase
complex (PDC). The case histories of 46 subjects were analyzed with regard to
diagnosis, clinical presentation and response to dichloroacetate.
Dichloroacetate decreased blood and cerebrospinal fluid lactate concentrations,
and was generally well tolerated. Dichloroacetate may be particularly effective
in children with PDC deficiency by stimulating residual enzyme activity and,
consequently, cellular energy metabolism. A controlled trial is needed to
determine the definitive role of dichloroacetate in the management of this
devastating disease.
Dichloroacetate studies in
rodents
Maternal exposure to high doses of trichloroethylene (TCE) and its oxidative
metabolites, trichloroacetic acid (TCA) and dichloroacetic acid, has been
implicated in eye malformations in fetal rats.
Dichloroacetate and neuropathy
Dichloroacetate causes toxic neuropathy in MELAS: a randomized, controlled
clinical trial. Neurology. 2006 Oct 10;67(7):1313; Comment on: Neurology. 2006
Feb 14;66(3):324-30.