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Review
. 2021 May 30;14(6):525.
doi: 10.3390/ph14060525.

Human Pluripotent Stem-Cell-Derived Models as a Missing Link in Drug Discovery and Development

Xiying Lin  1 , Jiayu Tang  1 , Yan-Ru Lou  1
Affiliations
Review

Human Pluripotent Stem-Cell-Derived Models as a Missing Link in Drug Discovery and Development

Xiying Lin et al. Pharmaceuticals (Basel). .

Abstract

Human pluripotent stem cells (hPSCs), including human embryonic stem cells (hESCs) and human-induced pluripotent stem cells (hiPSCs), have the potential to accelerate the drug discovery and development process. In this review, by analyzing each stage of the drug discovery and development process, we identified the active role of hPSC-derived in vitro models in phenotypic screening, target-based screening, target validation, toxicology evaluation, precision medicine, clinical trial in a dish, and post-clinical studies. Patient-derived or genome-edited PSCs can generate valid in vitro models for dissecting disease mechanisms, discovering novel drug targets, screening drug candidates, and preclinically and post-clinically evaluating drug safety and efficacy. With the advances in modern biotechnologies and developmental biology, hPSC-derived in vitro models will hopefully improve the cost-effectiveness and the success rate of drug discovery and development.

Keywords: drug development; drug discovery; human embryonic stem cells; human pluripotent stem cells; human-induced pluripotent stem cells.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
The workflow of drug discovery and development. Words in red font are stages that may involve hPSCs. ADME: absorption, distribution, metabolism, and excretion.
Figure 2
Figure 2
Types and possible applications of hPSC-derived models.
See this image and copyright information in PMC

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