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Updated: Jul 24, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Repurposing Two Old Friends to Fight Cancer: Caffeine and Statins
Derek W Stouth1, Paul F Lebeau1, Richard C Austin1
1Department of Medicine, Division of Nephrology, McMaster University, The Research Institute of St. Joe's Hamilton and The Hamilton Center for Kidney Research, Hamilton, Ontario, Canada.
Abstract:
Statins are a class of cholesterol-lowering drugs that inhibit 3-hydroxy-3-methylglutaryl-CoA reductase, the rate-limiting enzyme of the mevalonate pathway. Evidence suggests that certain cancers depend on the mevalonate pathway for growth and survival, and thus blocking the mevalonate pathway with statins may offer a viable therapeutic approach for treating cancer, or at least enhance the efficacy of existing cancer drugs. In this issue of Cancer Research, Tran and colleagues showed that caffeine works jointly with FOXM1 inhibition to enhance the antitumor activity of statins in neuroblastoma cells. They found that caffeine synergizes with statins by suppressing statin-induced feedback activation of the mevalonate pathway. Here, we reflect on the potential of combining caffeine and statin drugs as a strategy for potentiating anticancer activity. See related article by Tran et al., p. 2248.
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