Notch2 blockade enhances hematopoietic stem cell mobilization and homing

Haematologica. 2017 Oct;102(10):1785-1795. doi: 10.3324/haematol.2017.168674. Epub 2017 Jul 20.

Abstract

Despite use of newer approaches, some patients being considered for autologous hematopoietic cell transplantation (HCT) may only mobilize limited numbers of hematopoietic progenitor cells (HPCs) into blood, precluding use of the procedure, or being placed at increased risk of complications due to slow hematopoietic reconstitution. Developing more efficacious HPC mobilization regimens and strategies may enhance the mobilization process and improve patient outcome. Although Notch signaling is not essential for homeostasis of adult hematopoietic stem cells (HSCs), Notch-ligand adhesive interaction maintains HSC quiescence and niche retention. Using Notch receptor blocking antibodies, we report that Notch2 blockade, but not Notch1 blockade, sensitizes hematopoietic stem cells and progenitors (HSPCs) to mobilization stimuli and leads to enhanced egress from marrow to the periphery. Notch2 blockade leads to transient myeloid progenitor expansion without affecting HSC homeostasis and self-renewal. We show that transient Notch2 blockade or Notch2-loss in mice lacking Notch2 receptor lead to decreased CXCR4 expression by HSC but increased cell cycling with CXCR4 transcription being directly regulated by the Notch transcriptional protein RBPJ. In addition, we found that Notch2-blocked or Notch2-deficient marrow HSPCs show an increased homing to the marrow, while mobilized Notch2-blocked, but not Notch2-deficient stem cells and progenitors, displayed a competitive repopulating advantage and enhanced hematopoietic reconstitution. These findings suggest that blocking Notch2 combined with the current clinical regimen may further enhance HPC mobilization and improve engraftment during HCT.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Agents / therapeutic use
  • Bone Marrow Cells / drug effects
  • Bone Marrow Cells / metabolism
  • Cell Cycle / drug effects
  • Cell Cycle / genetics
  • Cell Differentiation / drug effects
  • Cell Differentiation / genetics
  • Cell Movement / drug effects
  • Cell Self Renewal / drug effects
  • Cell Self Renewal / genetics
  • Gene Expression Regulation / drug effects
  • Hematopoietic Stem Cell Mobilization* / methods
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / methods
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / drug effects*
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Mice
  • Mice, Transgenic
  • Receptor, Notch2 / antagonists & inhibitors*
  • Receptor, Notch2 / deficiency
  • Receptor, Notch2 / metabolism
  • Receptors, CXCR4 / genetics
  • Receptors, CXCR4 / metabolism
  • Signal Transduction / drug effects

Substances

  • Antineoplastic Agents
  • NOTCH2 protein, human
  • Receptor, Notch2
  • Receptors, CXCR4