PTPσ inhibitors promote hematopoietic stem cell regeneration

Nat Commun. 2019 Aug 14;10(1):3667. doi: 10.1038/s41467-019-11490-5.

Abstract

Receptor type protein tyrosine phosphatase-sigma (PTPσ) is primarily expressed by adult neurons and regulates neural regeneration. We recently discovered that PTPσ is also expressed by hematopoietic stem cells (HSCs). Here, we describe small molecule inhibitors of PTPσ that promote HSC regeneration in vivo. Systemic administration of the PTPσ inhibitor, DJ001, or its analog, to irradiated mice promotes HSC regeneration, accelerates hematologic recovery, and improves survival. Similarly, DJ001 administration accelerates hematologic recovery in mice treated with 5-fluorouracil chemotherapy. DJ001 displays high specificity for PTPσ and antagonizes PTPσ via unique non-competitive, allosteric binding. Mechanistically, DJ001 suppresses radiation-induced HSC apoptosis via activation of the RhoGTPase, RAC1, and induction of BCL-XL. Furthermore, treatment of irradiated human HSCs with DJ001 promotes the regeneration of human HSCs capable of multilineage in vivo repopulation. These studies demonstrate the therapeutic potential of selective, small-molecule PTPσ inhibitors for human hematopoietic regeneration.

Publication types

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

MeSH terms

  • Allosteric Regulation
  • Animals
  • Antimetabolites, Antineoplastic / pharmacology
  • Apoptosis / drug effects*
  • Apoptosis / radiation effects
  • Enzyme Inhibitors / pharmacology*
  • Fluorouracil / pharmacology
  • Hematopoietic Stem Cells / drug effects*
  • Hematopoietic Stem Cells / radiation effects
  • Humans
  • Mice
  • Radiation
  • Receptor-Like Protein Tyrosine Phosphatases, Class 2 / antagonists & inhibitors*
  • Regeneration / drug effects*
  • Regeneration / radiation effects
  • bcl-X Protein / drug effects
  • bcl-X Protein / metabolism
  • rac1 GTP-Binding Protein / drug effects
  • rac1 GTP-Binding Protein / metabolism
  • rho GTP-Binding Proteins / drug effects
  • rho GTP-Binding Proteins / metabolism

Substances

  • Antimetabolites, Antineoplastic
  • Enzyme Inhibitors
  • bcl-X Protein
  • Receptor-Like Protein Tyrosine Phosphatases, Class 2
  • rac1 GTP-Binding Protein
  • rho GTP-Binding Proteins
  • Fluorouracil