Drug discovery through stem cell-based organoid models

Adv Drug Deliv Rev. 2014 Apr;69-70:19-28. doi: 10.1016/j.addr.2014.02.006. Epub 2014 Feb 26.


The development of new drugs is currently a long and costly process in large part due to the failure of promising drug candidates identified in initial in vitro screens to perform as intended in vivo. New approaches to drug screening are being developed which focus on providing more biomimetic platforms. This review surveys this new generation of drug screening technologies, and provides an overview of recent developments in organoid culture systems which could afford previously unmatched fidelity for testing bioactivity and toxicity. The challenges inherent in such approaches will also be discussed, with a view towards bridging the gap between proof-of-concept studies and a wider implementation within the drug development community.

Keywords: 3D cell culture; Drug discovery; High-throughput screening; Organoid; Stem cell.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods
  • Drug Discovery / trends*
  • Drug Evaluation, Preclinical / methods
  • Drug Evaluation, Preclinical / trends
  • Drug-Related Side Effects and Adverse Reactions / diagnosis
  • Drug-Related Side Effects and Adverse Reactions / prevention & control
  • Humans
  • Organoids / cytology
  • Organoids / drug effects*
  • Organoids / physiology*
  • Stem Cells / drug effects*
  • Stem Cells / physiology*