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.,
Journal of Medicinal Chemistry
Department of Pediatrics

Zomer-van Ommen, D. D., Pukin, A. V., Fu, O. (Ou), Quarles Van Ufford, L.H.C. (Linda H.C.), Janssens, H., Beekman, J., & Pieters, R.J. (Roland J.). (2016). Functional characterization of cholera toxin inhibitors using human intestinal organoids. Journal of Medicinal Chemistry, 59(14), 6968–6972. doi:10.1021/acs.jmedchem.6b00770