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Review
. 2014 Mar 3;33(5):409-17.
doi: 10.1002/embj.201387098. Epub 2014 Feb 5.

iPS cells: a game changer for future medicine

Haruhisa Inoue  1 , Naoki Nagata, Hiromi Kurokawa, Shinya Yamanaka
Affiliations
Review

iPS cells: a game changer for future medicine

Haruhisa Inoue et al. EMBO J. .

Abstract

The induced pluripotent stem cell (iPSC) technology is instrumental in advancing the fields of disease modeling and cell transplantation. We herein discuss the various issues regarding disease modeling and cell transplantation presented in previous reports, and also describe new iPSC-based medicine including iPSC clinical trials. In such trials, iPSCs from patients can be used to predict drug responders/non-responders by analyzing the efficacy of the drug on iPSC-derived cells. They could also be used to stratify patients after actual clinical trials, including those with sporadic diseases, based on the drug responsiveness of each patient in the clinical trials. iPSC-derived cells can be used for the identification of response markers, leading to increased success rates in such trials. Since iPSCs can be used in micromedicine for drug discovery, and in macromedicine for actual clinical trials, their use would tightly connect both micro- and macromedicine. The use of iPSCs in disease modeling, cell transplantation, and clinical trials could therefore lead to significant changes in the future of medicine.

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Figures

Figure 1
Figure 1
iPSC technology contributes to ‘Disease modeling’, ‘Cell transplantation’, and ‘Clinical trial’. (A) Disease modeling using patient iPSCs for ‘Multi-omics’ and ‘Drug discovery’. (B) Instead of ‘Cell transplantation’, ‘Onsite reprogramming’ may be applied. (C) iPSCs from a large cohort of patients can be applied to ‘Clinical trial’ and ‘Patient stratification’.
Figure 2
Figure 2
iPSC medicine in drug development ranges from ‘Micromedicine’ to ‘Macromedicine’.
Figure 3
Figure 3
‘iPSC clinical trial’ for phase 0.5, 1.5, and 2.
Figure 4
Figure 4
Patient diagnoses in the past, present, and future.
See this image and copyright information in PMC

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