What Is Dichloroacetate?

Dichloroacetate (DCA) is the salt version of dichloroacetic acid, a compound sometimes created in the body as chlorine-containing drugs are broken down. This particular molecule works in the body to shut off the action of the protein pyruvate dehydrogenase kinase (PDK). PDK is an enzyme often found in certain cancer cells, and changes how these cells are able to use oxygen, by shutting down the usual pathways that process this important chemical. Consequently, there has been some medical interest in the use of DCA to combat cancer; this drug already has seen use in the treatment of some metabolic disorders.

Abnormal metabolic conditions can involve the body creating altered versions of enzymes that normally help it to survive. Lactic acidosis is one such disorder, which involves overactive copies of the PDK protein, leading to a buildup of lactate. By stopping the activity of PDK, dichloroacetate allows another protein, pyruvate dehydrogenase, to metabolize compounds like pyruvate in another manner. Some research has shown some evidence for symptom alleviation when DCA is used, but other studies have suggested that this drug could potentially be linked to serious side effects, such as nerve damage.

Cancer cells may be a possible target of dichloroacetate in therapy due to the same action of this enzyme. Through enhanced activity on the part of PDK, cancer cells can generate their own energy without the use of components called mitochondria. Normal cells do not use the same metabolic pathway to create energy, so in theory, healthy cells would be relatively unaffected through DCA treatment.

Some studies indicate that dichloroacetate could prove effective as a cancer therapy, although the research on this topic is still ongoing. One study, conducted at the University of Alberta, found that this drug did not damage healthy human cells that were cultivated in a laboratory, but that several types of cancer cells perished after they were exposed to DCA. Shutting off the PDK pathway reactivated the mitochondria in these cells, triggering a self-destruct mechanism in these cells.

Limited studies in humans have shown some evidence for the effectiveness of dichloroacetate. Individuals with cancer that have taken this drug have sometimes shown decreased tumor sizes, although there has not yet been enough research to determine whether DCA prolongs the lifespan of cancer patients. Studies have shown, however, that this medication tends to have a minimal amount of side effects for most people that have taken it. Some medical professionals have cautioned that more side effects could become apparent as more research on this compound is conducted, and that people taking DCA to self-medicate for cancer could potentially be risking their health.