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Essai de médicaments antitumoraux sur des humains volontaires

Published online : 28 March 2007; | doi:10.1038/446474a / http://www.nature.com/news/2007/070326/full/446474a.html

Cancer patients opt for unapproved drug

Internet trade pre-empts clinical trial.

Helen Pearson

An experimental cancer drug shrinks tumours in rats with no apparent side effects. The scientists behind the study plan to do a clinical trial in humans, but it could take years to complete. Meanwhile, dying patients begin taking the unapproved drug and collect their results on the web. Both groups desperately want to save lives: but which is the right route to follow?

This scenario has been playing out in recent weeks for a compound called dichloroacetate (DCA). It taps into long-running issues about whether terminally ill patients should be able to get access to drugs that have not yet had formal approval. Researchers fear that those taking the drug could suffer unanticipated side effects; patients argue they don't have the luxury of waiting for clinical trials to find out.







In January this year, Evangelos Michelakis at the University of Alberta in Edmonton, Canada, and his colleagues reported that DCA has seemingly remarkable anticancer properties (S. Bonnet et al. Cancer Cell 11, 37-51; 2007). DCA is a small molecule that blocks an enzyme in mitochondria - the energy-production centres in cells - causing more glucose to be metabolized in the mitochondria rather than by a different pathway in the cytoplasm. The compound has been in clinical trials for years as a treatment for certain mitochondrial diseases, but it has not yet been approved.

Mitochondria also control cell suicide, and Michelakis wondered whether cancer cells were suppressing these cellular structures to prevent the cells from dying - and so thought DCA might reactivate them. When his team gave DCA to rats that were growing human lung tumours, the tumours stopped growing within a week, and three months later were half the size of those in untreated animals. Other experimental drugs have had similar effects. But DCA stands out because it seems to leave healthy cells untouched, has been relatively safe in human trials, can be taken by mouth and easily penetrates tissues. "If there were a magic bullet," wrote Newsweek about the discovery, "it might be something like dichloroacetate."


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