Immunotherapy-based targeting of MSLN+ activated portal fibroblasts is a strategy for treatment of cholestatic liver fibrosis

Proc Natl Acad Sci U S A. 2021 Jul 20;118(29):e2101270118. doi: 10.1073/pnas.2101270118.

Abstract

We investigated the role of mesothelin (Msln) and thymocyte differentiation antigen 1 (Thy1) in the activation of fibroblasts across multiple organs and demonstrated that Msln-/- mice are protected from cholestatic fibrosis caused by Mdr2 (multidrug resistance gene 2) deficiency, bleomycin-induced lung fibrosis, and UUO (unilateral urinary obstruction)-induced kidney fibrosis. On the contrary, Thy1-/- mice are more susceptible to fibrosis, suggesting that a Msln-Thy1 signaling complex is critical for tissue fibroblast activation. A similar mechanism was observed in human activated portal fibroblasts (aPFs). Targeting of human MSLN+ aPFs with two anti-MSLN immunotoxins killed fibroblasts engineered to express human mesothelin and reduced collagen deposition in livers of bile duct ligation (BDL)-injured mice. We provide evidence that antimesothelin-based therapy may be a strategy for treatment of parenchymal organ fibrosis.

Keywords: activated portal fibroblasts; cholestatic fibrosis; mesothelin.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Cholestasis / drug therapy*
  • Cholestasis / genetics
  • Cholestasis / immunology
  • Collagen / immunology
  • Fibroblasts / drug effects
  • Fibroblasts / immunology*
  • Humans
  • Immunotherapy*
  • Immunotoxins / administration & dosage
  • Liver Cirrhosis / drug therapy*
  • Liver Cirrhosis / genetics
  • Liver Cirrhosis / immunology
  • Mesothelin / genetics
  • Mesothelin / immunology
  • Mice
  • Thy-1 Antigens / genetics
  • Thy-1 Antigens / immunology

Substances

  • Immunotoxins
  • Msln protein, mouse
  • Thy-1 Antigens
  • Collagen
  • Mesothelin