Human iPSC modeling of heart disease for drug development

Cell Chem Biol. 2021 Mar 18;28(3):271-282. doi: 10.1016/j.chembiol.2021.02.016.


Human induced pluripotent stem cells (hiPSCs) have emerged as a promising platform for pharmacogenomics and drug development. In cardiology, they make it possible to produce unlimited numbers of patient-specific human cells that reproduce hallmark features of heart disease in the culture dish. Their potential applications include the discovery of mechanism-specific therapeutics, the evaluation of safety and efficacy in a human context before a drug candidate reaches patients, and the stratification of patients for clinical trials. Although this new technology has the potential to revolutionize drug discovery, translational hurdles have hindered its widespread adoption for pharmaceutical development. Here we discuss recent progress in overcoming these hurdles that should facilitate the use of hiPSCs to develop new medicines and individualize therapies for heart disease.

Keywords: HTS; cardiomyocyte; disease modeling; drug attrition; drug discovery; heart disease; hiPSC; screen.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Cardiovascular Agents / chemical synthesis
  • Cardiovascular Agents / chemistry
  • Cardiovascular Agents / pharmacology*
  • Drug Development*
  • Heart Diseases / drug therapy*
  • Heart Diseases / pathology
  • Humans
  • Induced Pluripotent Stem Cells / drug effects*


  • Cardiovascular Agents