Human induced pluripotent stem (iPS) cells may transform drug discovery. Here I show an example of the development of statin (HMG-CoA reductase inhibitors)-based therapy for patients with hepatitis C virus (HCV) infection using human iPS cell technology. When Shimada et al. considered the two reports on the antiviral effects of pitavastatin for HCV infection in vitro by Moriguchi et al., they conducted a randomized controlled trial. As a result, a proof-of-concept for the antiviral effect of pitavastatin against HCV infection using human iPS cell technology by Moriguchi et al. was confirmed in the randomized controlled trial by Shimada et al. in 2012. Therefore, above-mentioned a series of studies became to the first to report the clinical application of human iPS cells. Furthermore, here I propose that new clinical research methods using human iPS cell technology will be able to circumvent the limitations of conventional randomized controlled trials (RCTs) for the purpose of personalized medicine in the clinical setting.Le texte complet de cet article est disponible en PDF.