Inhibition of the Bcl-xL deamidation pathway in myeloproliferative disorders

N Engl J Med. 2008 Dec 25;359(26):2778-89. doi: 10.1056/NEJMoa0804953.

Abstract

Background: The myeloproliferative disorders are clonal disorders with frequent somatic gain-of-function alterations affecting tyrosine kinases. In these diseases, there is an increase in DNA damage and a risk of progression to acute leukemia. The molecular mechanisms in myeloproliferative disorders that prevent apoptosis induced by damaged DNA are obscure.

Methods: We searched for abnormalities of the proapoptotic Bcl-x(L) deamidation pathway in primary cells from patients with chronic myeloid leukemia (CML) or polycythemia vera, myeloproliferative disorders associated with the BCR-ABL fusion kinase and the Janus tyrosine kinase 2 (JAK2) V617F mutation, respectively.

Results: The Bcl-x(L) deamidation pathway was inhibited in myeloid cells, but not T cells, in patients with CML or polycythemia vera. DNA damage did not increase levels of the amiloride-sensitive sodium-hydrogen exchanger isoform 1 (NHE-1), intracellular pH, Bcl-x(L) deamidation, and apoptosis. Inhibition of the pathway was reversed by enforced alkalinization or overexpression of NHE-1, leading to a restoration of apoptosis. In patients with CML, the pathway was blocked in CD34+ progenitor cells and mature myeloid cells. Imatinib or JAK2 inhibitors reversed inhibition of the pathway in cells from patients with CML and polycythemia vera, respectively, but not in cells from a patient with resistance to imatinib because of a mutation in the BCR-ABL kinase domain.

Conclusions: BCR-ABL and mutant JAK2 inhibit the Bcl-x(L) deamidation pathway and the apoptotic response to DNA damage in primary cells from patients with CML or polycythemia vera.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Apoptosis
  • Cation Transport Proteins / genetics
  • Cell Line, Tumor
  • DNA Damage* / genetics
  • Deamination
  • Fusion Proteins, bcr-abl / genetics
  • Fusion Proteins, bcr-abl / physiology
  • Gene Transfer Techniques
  • Genes, abl / genetics
  • Humans
  • Janus Kinase 2 / genetics
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / blood
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / genetics*
  • Leukocytes, Mononuclear
  • Myeloproliferative Disorders / blood
  • Myeloproliferative Disorders / genetics
  • Polycythemia Vera / blood
  • Polycythemia Vera / genetics*
  • Protein-Tyrosine Kinases / genetics
  • Protein-Tyrosine Kinases / physiology
  • Signal Transduction / genetics
  • Sodium-Hydrogen Exchanger 1
  • Sodium-Hydrogen Exchangers / genetics
  • bcl-X Protein / genetics
  • bcl-X Protein / physiology*

Substances

  • Cation Transport Proteins
  • SLC9A1 protein, human
  • Sodium-Hydrogen Exchanger 1
  • Sodium-Hydrogen Exchangers
  • bcl-X Protein
  • Protein-Tyrosine Kinases
  • Fusion Proteins, bcr-abl
  • Janus Kinase 2