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Targeting the pregnane X receptor using microbial metabolite mimicry
Journal article   Open access   Peer reviewed

Targeting the pregnane X receptor using microbial metabolite mimicry

Zdeněk Dvořák, Felix Kopp, Cait M Costello, Jazmin S Kemp, Hao Li, Aneta Vrzalová, Martina Štěpánková, Iveta Bartoňková, Eva Jiskrová, Karolína Poulíková, …
EMBO molecular medicine, v 12(4), pp e11621-n/a
07 Apr 2020
PMID: 32153125
url
https://doi.org/10.15252/emmm.201911621View
Published, Version of Record (VoR) Open

Abstract

Chemical Biology Digestive System drugs Immunology microbial metabolite mimics pregnane X receptor therapy
The human PXR (pregnane X receptor), a master regulator of drug metabolism, has essential roles in intestinal homeostasis and abrogating inflammation. Existing PXR ligands have substantial off‐target toxicity. Based on prior work that established microbial (indole) metabolites as PXR ligands, we proposed microbial metabolite mimicry as a novel strategy for drug discovery that allows exploiting previously unexplored parts of chemical space. Here, we report functionalized indole derivatives as first‐in‐class non‐cytotoxic PXR agonists as a proof of concept for microbial metabolite mimicry. The lead compound, FKK 6 (Felix Kopp Kortagere 6), binds directly to PXR protein in solution, induces PXR ‐specific target gene expression in cells, human organoids, and mice. FKK 6 significantly represses pro‐inflammatory cytokine production cells and abrogates inflammation in mice expressing the human PXR gene. The development of FKK 6 demonstrates for the first time that microbial metabolite mimicry is a viable strategy for drug discovery and opens the door to underexploited regions of chemical space. This study demonstrates that microbial metabolite mimicry can expand the chemical space in drug discovery. Chemical mimics of microbial indoles interacting with a host nuclear receptor provides a novel and non‐toxic therapeutic approach for treating inflammatory conditions of the intestine.

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Collaboration types
Domestic collaboration
International collaboration
Web of Science research areas
Medicine, Research & Experimental
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