Notch pathway activation targets AML-initiating cell homeostasis and differentiation

J Exp Med. 2013 Feb 11;210(2):301-19. doi: 10.1084/jem.20121484. Epub 2013 Jan 28.

Abstract

Notch signaling pathway activation is known to contribute to the pathogenesis of a spectrum of human malignancies, including T cell leukemia. However, recent studies have implicated the Notch pathway as a tumor suppressor in myeloproliferative neoplasms and several solid tumors. Here we report a novel tumor suppressor role for Notch signaling in acute myeloid leukemia (AML) and demonstrate that Notch pathway activation could represent a therapeutic strategy in this disease. We show that Notch signaling is silenced in human AML samples, as well as in AML-initiating cells in an animal model of the disease. In vivo activation of Notch signaling using genetic Notch gain of function models or in vitro using synthetic Notch ligand induces rapid cell cycle arrest, differentiation, and apoptosis of AML-initiating cells. Moreover, we demonstrate that Notch inactivation cooperates in vivo with loss of the myeloid tumor suppressor Tet2 to induce AML-like disease. These data demonstrate a novel tumor suppressor role for Notch signaling in AML and elucidate the potential therapeutic use of Notch receptor agonists in the treatment of this devastating leukemia.

Publication types

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

MeSH terms

  • Animals
  • Cell Differentiation
  • Cell Survival
  • DNA-Binding Proteins / metabolism
  • Dioxygenases
  • Disease Models, Animal
  • Gene Silencing
  • Homeostasis
  • Humans
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / metabolism*
  • Leukemia, Myeloid, Acute / pathology*
  • Leukemia, Myeloid, Acute / therapy
  • Ligands
  • Mice
  • Mice, Transgenic
  • Mutation
  • Proto-Oncogene Proteins / metabolism
  • Receptors, Notch / agonists
  • Receptors, Notch / metabolism*
  • Signal Transduction
  • Tumor Suppressor Proteins / metabolism

Substances

  • DNA-Binding Proteins
  • Ligands
  • Proto-Oncogene Proteins
  • Receptors, Notch
  • Tumor Suppressor Proteins
  • Dioxygenases
  • TET2 protein, human