A cationic-independent mannose 6-phosphate receptor inhibitor (PXS64) ameliorates kidney fibrosis by inhibiting activation of transforming growth factor-β1

PLoS One. 2015 Feb 6;10(2):e0116888. doi: 10.1371/journal.pone.0116888. eCollection 2015.

Abstract

The activity of transforming growth factor-β1 (TGF-β1) is regulated by its conversion from the latent to the active form. We have previously shown that the conversion is at least in part mediated by the cationic-independent mannose 6-phosphate receptor (CI-M6PR), as the CI-M6PR inhibitor, PXS-25 has anti-fibrotic properties in human kidney tubular (HK-2) cells under high glucose conditions. However, its clinical use is limited by low bioavailability. Our aim was to determine the effects of PXS64, a pro-drug of PXS25, in in vitro and in vivo models of renal fibrosis. HK-2 cells were exposed to latent TGFβ1+/- PXS64 for 48 hours. The mRNA and protein levels of pro-fibrotic and pro-inflammatory markers were determined. A 7 day unilateral ureteric obstruction (UUO) model was used and the following experimental groups were studied: (i) Sham operated, (ii) UUO, (iii) UUO + telmisartan (iv) UUO + PSX64. HK-2 cells exposed to PXS64 reduced TGFβ mediated effects on collagen IV, fibronectin, macrophage chemotactic protein-1 (MCP-1) and phospho-smad2 protein expression, consistent with inhibition of the conversion of latent to active TGF-β1. PXS 64 treated UUO mice had a lower tubulointerstitial fibrosis index, collagen IV and fibronectin protein and mRNA expression when compared to untreated UUO mice. In addition, these animals had lower MCP-1 mRNA expression, reduced inflammarory cell infiltrate, as indicated by fewer CD45, F4/80 positive cells, and reduced phospho-Smad2 protein expression when compared to untreated UUO animals. Our data demonstrates that PSX64 is an effective anti-fibrotic agent by inhibiting the activation of latent TGF-β1.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cells, Cultured
  • Disease Models, Animal
  • Fibrosis / drug therapy*
  • Fibrosis / genetics
  • Fibrosis / metabolism
  • Fibrosis / pathology
  • Gene Expression Regulation / drug effects
  • Humans
  • Kidney / drug effects
  • Kidney / pathology*
  • Kidney Tubules / cytology
  • Kidney Tubules / drug effects*
  • Kidney Tubules / metabolism
  • Male
  • Mannosides / pharmacology*
  • Mice, Inbred C57BL
  • Neutrophils / drug effects
  • Organophosphonates / pharmacology*
  • Prodrugs / pharmacology
  • Smad2 Protein / metabolism
  • Transforming Growth Factor beta / metabolism*
  • Transforming Growth Factor beta / pharmacology
  • Ureteral Obstruction / pathology

Substances

  • Mannosides
  • Organophosphonates
  • PXS64 compound
  • Prodrugs
  • SMAD2 protein, human
  • Smad2 Protein
  • Transforming Growth Factor beta

Grants and funding

The study is funded by the Australian Research Council (ARC) industry partnership linkage grant. None the co-authors has a financial disclosure. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.