Kynurenine-3-monooxygenase inhibition prevents multiple organ failure in rodent models of acute pancreatitis

Nat Med. 2016 Feb;22(2):202-9. doi: 10.1038/nm.4020. Epub 2016 Jan 11.

Abstract

Acute pancreatitis (AP) is a common and devastating inflammatory condition of the pancreas that is considered to be a paradigm of sterile inflammation leading to systemic multiple organ dysfunction syndrome (MODS) and death. Acute mortality from AP-MODS exceeds 20% (ref. 3), and the lifespans of those who survive the initial episode are typically shorter than those of the general population. There are no specific therapies available to protect individuals from AP-MODS. Here we show that kynurenine-3-monooxygenase (KMO), a key enzyme of tryptophan metabolism, is central to the pathogenesis of AP-MODS. We created a mouse strain that is deficient for Kmo (encoding KMO) and that has a robust biochemical phenotype that protects against extrapancreatic tissue injury to the lung, kidney and liver in experimental AP-MODS. A medicinal chemistry strategy based on modifications of the kynurenine substrate led to the discovery of the oxazolidinone GSK180 as a potent and specific inhibitor of KMO. The binding mode of the inhibitor in the active site was confirmed by X-ray co-crystallography at 3.2 Å resolution. Treatment with GSK180 resulted in rapid changes in the levels of kynurenine pathway metabolites in vivo, and it afforded therapeutic protection against MODS in a rat model of AP. Our findings establish KMO inhibition as a novel therapeutic strategy in the treatment of AP-MODS, and they open up a new area for drug discovery in critical illness.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Disease
  • Animals
  • Benzoxazoles / pharmacology*
  • Chromatography, Liquid
  • Crystallography, X-Ray
  • Disease Models, Animal
  • HEK293 Cells
  • Hepatocytes / metabolism
  • Humans
  • In Vitro Techniques
  • Kidney / metabolism
  • Kidney / pathology
  • Kynurenine 3-Monooxygenase / antagonists & inhibitors*
  • Kynurenine 3-Monooxygenase / genetics
  • Lung / metabolism
  • Lung / pathology
  • Mice
  • Mice, Knockout
  • Multiple Organ Failure / etiology
  • Multiple Organ Failure / genetics*
  • Multiple Organ Failure / pathology
  • Oxazolidinones / pharmacology*
  • Pancreas / metabolism
  • Pancreas / pathology
  • Pancreatitis / complications
  • Pancreatitis / genetics*
  • Pancreatitis / pathology
  • Propionates / pharmacology*
  • RNA, Messenger / metabolism*
  • Rats
  • Tandem Mass Spectrometry
  • Tryptophan / metabolism

Substances

  • Benzoxazoles
  • GSK180
  • Oxazolidinones
  • Propionates
  • RNA, Messenger
  • Tryptophan
  • Kynurenine 3-Monooxygenase