Clinical Trials in a Dish: The Potential of Pluripotent Stem Cells to Develop Therapies for Neurodegenerative Diseases

Annu Rev Pharmacol Toxicol. 2016;56:489-510. doi: 10.1146/annurev-pharmtox-010715-103548. Epub 2015 Oct 28.


Neurodegenerative diseases are a leading cause of death. No disease-modifying therapies are available, and preclinical animal model data have routinely failed to translate into success for therapeutics. Induced pluripotent stem cell (iPSC) biology holds great promise for human in vitro disease modeling because these cells can give rise to any cell in the human brain and display phenotypes specific to neurodegenerative diseases previously identified in postmortem and clinical samples. Here, we explore the potential and caveats of iPSC technology as a platform for drug development and screening, and the future potential to use large cohorts of disease-bearing iPSCs to perform clinical trials in a dish.

Keywords: clinical trials; drug development; human pluripotent stem cells; neurodegeneration.

Publication types

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

MeSH terms

  • Animals
  • Brain / physiopathology
  • Clinical Trials as Topic
  • Humans
  • Neurodegenerative Diseases / physiopathology*
  • Pluripotent Stem Cells / physiology*