Biological basis for efficacy of activin receptor ligand traps in myelodysplastic syndromes

J Clin Invest. 2020 Feb 3;130(2):582-589. doi: 10.1172/JCI133678.

Abstract

Signaling by the TGF-β superfamily is important in the regulation of hematopoiesis and is dysregulated in myelodysplastic syndromes (MDSs), contributing to ineffective hematopoiesis and clinical cytopenias. TGF-β, activins, and growth differentiation factors exert inhibitory effects on red cell formation by activating canonical SMAD2/3 pathway signaling. In this Review, we summarize evidence that overactivation of SMAD2/3 signaling pathways in MDSs causes anemia due to impaired erythroid maturation. We also describe the basis for biological activity of activin receptor ligand traps, novel fusion proteins such as luspatercept that are promising as erythroid maturation agents to alleviate anemia and related comorbidities in MDSs and other conditions characterized by impaired erythroid maturation.

Publication types

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

MeSH terms

  • Activin Receptors / metabolism*
  • Activin Receptors, Type II / therapeutic use*
  • Animals
  • Erythrocytes / metabolism*
  • Erythrocytes / pathology
  • Erythropoiesis / drug effects*
  • Humans
  • Immunoglobulin Fc Fragments / therapeutic use*
  • Myelodysplastic Syndromes* / drug therapy
  • Myelodysplastic Syndromes* / metabolism
  • Myelodysplastic Syndromes* / pathology
  • Recombinant Fusion Proteins / therapeutic use*
  • Signal Transduction / drug effects*
  • Smad2 Protein / metabolism
  • Smad3 Protein / metabolism

Substances

  • Immunoglobulin Fc Fragments
  • Recombinant Fusion Proteins
  • SMAD2 protein, human
  • SMAD3 protein, human
  • Smad2 Protein
  • Smad3 Protein
  • luspatercept
  • Activin Receptors
  • Activin Receptors, Type II