Patients Using Unapproved Cancer Agent

OTTAWA, Canada—University of Alberta researchers, led by Evangelos Michelakis, MD, have shown that dichloroacetate (DCA), added to the drinking water of nude athymic rats implanted with human lung cancer cells (A549), significantly reduced tumor size, compared with controls, and was associated with increased apoptosis and decreased proliferation. Further, there were no toxic effects, as measured by several blood tests (Cancer Cell 11:37-51, 2007).

DCA, a small molecule that targets mitochondria, has long been used to treat lactic acidosis and inherited mitochondrial diseases, but is not FDA approved. It appears to work in cancer by reactivating the mitochondria of cancer cells, thus inducing apoptosis. Because its structure cannot be patented, DCA has not attracted commercial development. But it has attracted the attention of cancer patients. According to Nature (March 29, 2007, p. 474), Jim Tassano and a chemist friend synthesized the compound and are selling it via the web (labeled for veterinary use). Another website (thedcasite.com) provides patients with information on DCA and a place to report their results. "Any way you look at this, it's a negative development," said Dr. Michelakis. His group is planning a clinical trial despite lack of industry support.