A controversial generic drug can prevent tumor growth in patients with an aggressive form of brain cancer, researchers reported today in the journal Science Translational Medicine.
The compound — called dichloroacetate, or DCA — could prove to be a cheap and effective way of treating brain cancer if the findings pan out in larger studies. But study author Evangelos Michelakis warned cancer patients not to order the chemical on the Internet and start self-medicating on the back of his small phase 1 trial.
Three years ago, Michelakis and his colleagues at the University of Alberta showed that the off-patent, and thus inexpensive, small molecule killed cancer cells in vitro and in a rat model of the disease by inhibiting the enzyme pyruvate dehydrogenase kinase within mitochondria to block cell metabolism.
Buoyed by those findings, a husband-and-wife team in Toronto started administering the compound to cancer patients in their clinic, even though it had never been clinically tested nor received regulatory approval. Last week, a Canadian man was also convicted in a Phoenix court for peddling sugar and starch purported to be DCA through a now defunct website.
These and other rogue treatments led New Scientist to editorialize that DCA “remains experimental, never yet properly tested in a person with cancer. People who self-administer the drug are taking a very long shot and, unlikely as it may sound, could even make their health worse.”
As an off-patent drug, there was little financial incentive to investigate the compound further. But Michelakis amassed $200,000 from private donors and has now tested DCA on five people with a type of brain cancer known as glioblastoma. He and his team tracked the patients for over a year and used brain imaging scans to show that the tumors shrank or stopped growing in four of the subjects.
DCA is current not approved for treating cancer by the US Food and Drug Administration. Were it given the go-ahead, however, Michelakis estimates that a standard treatment could be manufactured for less than $100 per year.
Image by pasukaru76 via Flickr Creative Commons