I received a question on the discussion forum, in the setting of a lot of internet discussion, about an an agent called dichloroacetate, or DCA, as a potential anticancer therapy. This excitement is based on a study out of the University of Alberta in Canada, that appeared in the journal Cancer Cell (article here). This is a very novel molecular therapy approach, and one about which I don’t have a lot of insight yet, but it does provide an opportunity to discuss the gap between a promising result in preclinical studies and a proven anticancer therapy.

DCA is an unpatented small molecule that has been used for decades to treat children with rare molecular disorders associated with mitochondrial problems, but it’s not a drug that I have had occasion to use. Mitochondria are a component of every cell that create energy for the cell, and mitochondrial disorders have been known to be seen with cancer but have been believed to be an effect rather than a key contributing cause of the cancer process.

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The idea is basically that cancer cells suppress their mitochondria, making the cells resistant to apoptosis, or the normal process of programmed cell death, a sort of self-destruct program built into all cells of the body for certain normal developmental processes but also as a defense mechanism in case the cells mutate too much to work properly. The work described in the paper demonstrated that multiple types of cancer cells in test tube (in vitro) and animal models could be suppressed and killed after DCA restored the normal ability of mitochondria and led to apoptosis that destroyed cancer cells. This research suggests it would have no effect on normal non-cancer cells, so it is believed that DCA would not be assocaited with significant side effects. Overall, these results were considered very encouraging for a drug that it inexpensive and orally available. Continue reading →