Activin-like kinase 3 is important for kidney regeneration and reversal of fibrosis

Nat Med. 2012 Feb 5;18(3):396-404. doi: 10.1038/nm.2629.

Abstract

Molecules associated with the transforming growth factor β (TGF-β) superfamily, such as bone morphogenic proteins (BMPs) and TGF-β, are key regulators of inflammation, apoptosis and cellular transitions. Here we show that the BMP receptor activin-like kinase 3 (Alk3) is elevated early in diseased kidneys after injury. We also found that its deletion in the tubular epithelium leads to enhanced TGF-β1-Smad family member 3 (Smad3) signaling, epithelial damage and fibrosis, suggesting a protective role for Alk3-mediated signaling in the kidney. A structure-function analysis of the BMP-Alk3-BMP receptor, type 2 (BMPR2) ligand-receptor complex, along with synthetic organic chemistry, led us to construct a library of small peptide agonists of BMP signaling that function through the Alk3 receptor. One such peptide agonist, THR-123, suppressed inflammation, apoptosis and the epithelial-to-mesenchymal transition program and reversed established fibrosis in five mouse models of acute and chronic renal injury. THR-123 acts specifically through Alk3 signaling, as mice with a targeted deletion for Alk3 in their tubular epithelium did not respond to therapy with THR-123. Combining THR-123 and the angiotensin-converting enzyme inhibitor captopril had an additive therapeutic benefit in controlling renal fibrosis. Our studies show that BMP signaling agonists constitute a new line of therapeutic agents with potential utility in the clinic to induce regeneration, repair and reverse established fibrosis.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Angiotensin-Converting Enzyme Inhibitors / pharmacology
  • Animals
  • Apoptosis / genetics
  • Bone Morphogenetic Protein Receptors / genetics
  • Bone Morphogenetic Protein Receptors / metabolism*
  • Bone Morphogenetic Protein Receptors, Type I / genetics
  • Bone Morphogenetic Protein Receptors, Type I / metabolism
  • Bone Morphogenetic Protein Receptors, Type II / genetics
  • Bone Morphogenetic Protein Receptors, Type II / metabolism
  • Bone Morphogenetic Proteins / agonists*
  • Bone Morphogenetic Proteins / metabolism
  • Captopril / pharmacology
  • Diabetic Nephropathies / metabolism
  • Diabetic Nephropathies / pathology
  • Epithelial-Mesenchymal Transition
  • Fibrosis / metabolism
  • Inflammation / genetics
  • Inflammation / metabolism
  • Kidney / injuries*
  • Kidney / metabolism*
  • Kidney Tubules / metabolism
  • Mice
  • Peptide Library
  • Peptides / chemical synthesis
  • Peptides / metabolism*
  • Peptides / pharmacokinetics
  • Rats
  • Rats, Sprague-Dawley
  • Regeneration / genetics*
  • Signal Transduction
  • Smad3 Protein / genetics
  • Smad3 Protein / metabolism*
  • Structure-Activity Relationship
  • Transforming Growth Factor beta / genetics
  • Transforming Growth Factor beta / metabolism*

Substances

  • Angiotensin-Converting Enzyme Inhibitors
  • Bone Morphogenetic Proteins
  • Peptide Library
  • Peptides
  • Smad3 Protein
  • Smad3 protein, mouse
  • Transforming Growth Factor beta
  • Captopril
  • Bmpr2 protein, mouse
  • Bone Morphogenetic Protein Receptors
  • Bone Morphogenetic Protein Receptors, Type I
  • Bone Morphogenetic Protein Receptors, Type II