CYT387, a novel JAK2 inhibitor, induces hematologic responses and normalizes inflammatory cytokines in murine myeloproliferative neoplasms

Blood. 2010 Jun 24;115(25):5232-40. doi: 10.1182/blood-2009-05-223727. Epub 2010 Apr 12.

Abstract

Activating alleles of Janus kinase 2 (JAK2) such as JAK2(V617F) are central to the pathogenesis of myeloproliferative neoplasms (MPN), suggesting that small molecule inhibitors targeting JAK2 may be therapeutically useful. We have identified an aminopyrimidine derivative (CYT387), which inhibits JAK1, JAK2, and tyrosine kinase 2 (TYK2) at low nanomolar concentrations, with few additional targets. Between 0.5 and 1.5muM CYT387 caused growth suppression and apoptosis in JAK2-dependent hematopoietic cell lines, while nonhematopoietic cell lines were unaffected. In a murine MPN model, CYT387 normalized white cell counts, hematocrit, spleen size, and restored physiologic levels of inflammatory cytokines. Despite the hematologic responses and reduction of the JAK2(V617F) allele burden, JAK2(V617F) cells persisted and MPN recurred upon cessation of treatment, suggesting that JAK2 inhibitors may be unable to eliminate JAK2(V617F) cells, consistent with preliminary results from clinical trials of JAK2 inhibitors in myelofibrosis. While the clinical benefit of JAK2 inhibitors may be substantial, not the least due to reduction of inflammatory cytokines and symptomatic improvement, our data add to increasing evidence that kinase inhibitor monotherapy of malignant disease is not curative, suggesting a need for drug combinations to optimally target the malignant cells.

Publication types

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

MeSH terms

  • Animals
  • Apoptosis / drug effects*
  • Apoptosis / genetics
  • Apoptosis / immunology
  • Benzamides / pharmacology*
  • Cell Line, Tumor
  • Cytokines / genetics
  • Cytokines / immunology
  • Cytokines / metabolism
  • Disease Models, Animal
  • Drug Screening Assays, Antitumor
  • Hematologic Neoplasms / drug therapy*
  • Hematologic Neoplasms / enzymology
  • Hematologic Neoplasms / genetics
  • Hematologic Neoplasms / immunology
  • Hematopoiesis / drug effects*
  • Hematopoiesis / genetics
  • Hematopoiesis / immunology
  • Janus Kinase 1 / antagonists & inhibitors
  • Janus Kinase 1 / genetics
  • Janus Kinase 1 / immunology
  • Janus Kinase 1 / metabolism
  • Janus Kinase 2 / antagonists & inhibitors*
  • Janus Kinase 2 / genetics
  • Janus Kinase 2 / immunology
  • Janus Kinase 2 / metabolism
  • Mice
  • Mice, Inbred BALB C
  • Mutation, Missense
  • Myeloproliferative Disorders / drug therapy*
  • Myeloproliferative Disorders / enzymology
  • Myeloproliferative Disorders / genetics
  • Myeloproliferative Disorders / immunology
  • Protein Kinase Inhibitors / pharmacology*
  • Pyrimidines / pharmacology*

Substances

  • Benzamides
  • Cytokines
  • Protein Kinase Inhibitors
  • Pyrimidines
  • N-(cyanomethyl)-4-(2-((4-(4-morpholinyl)phenyl)amino)-4-pyrimidinyl)benzamide
  • Jak1 protein, mouse
  • Jak2 protein, mouse
  • Janus Kinase 1
  • Janus Kinase 2