Functional Characterization of Cholera Toxin Inhibitors Using Human Intestinal Organoids

J Med Chem. 2016 Jul 28;59(14):6968-72. doi: 10.1021/acs.jmedchem.6b00770. Epub 2016 Jul 7.

Abstract

Preclinical drug testing in primary human cell models that recapitulate disease can significantly reduce animal experimentation and time-to-the-clinic. We used intestinal organoids to quantitatively study the potency of multivalent cholera toxin inhibitors. The method enabled the determination of IC50 values over a wide range of potencies (15 pM to 9 mM). The results indicate for the first time that an organoid-based swelling assay is a useful preclinical method to evaluate inhibitor potencies of drugs that target pathogen-derived toxins.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antitoxins / chemistry
  • Antitoxins / pharmacology*
  • Cholera Toxin / antagonists & inhibitors*
  • Dose-Response Relationship, Drug
  • Humans
  • Intestines / drug effects*
  • Molecular Structure
  • Organoids / drug effects*
  • Structure-Activity Relationship

Substances

  • Antitoxins
  • Cholera Toxin