Persistence of leukemia-initiating cells in a conditional knockin model of an imatinib-responsive myeloproliferative disorder

Cancer Cell. 2009 Aug 4;16(2):137-48. doi: 10.1016/j.ccr.2009.06.007.

Abstract

Despite remarkable responses to the tyrosine kinase inhibitor imatinib, CML patients are rarely cured by this therapy perhaps due to imatinib refractoriness of leukemia-initiating cells (LICs). Evidence for this is limited because of poor engraftment of human CML-LICs in NOD-SCID mice and nonphysiologic expression of oncogenes in retroviral transduction mouse models. To address these challenges, we generated mice bearing conditional knockin alleles of two human oncogenes: HIP1/PDGFbetaR (H/P) and AML1-ETO (A/E). Unlike retroviral transduction, physiologic expression of H/P or A/E individually failed to induce disease, but coexpression of both H/P and A/E led to rapid onset of a fully penetrant, myeloproliferative disorder, indicating cooperativity between these two alleles. Although imatinib dramatically decreased disease burden, LICs persisted, demonstrating imatinib refractoriness of LICs.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / therapeutic use*
  • Benzamides
  • Core Binding Factor Alpha 2 Subunit / genetics*
  • DNA-Binding Proteins / genetics*
  • Disease Models, Animal
  • Drug Resistance, Neoplasm / genetics
  • Gene Knock-In Techniques
  • Genotype
  • Hematopoietic Stem Cells / drug effects
  • Hematopoietic Stem Cells / metabolism
  • Hematopoietic Stem Cells / pathology
  • Humans
  • Imatinib Mesylate
  • Leukemia, Myelomonocytic, Chronic / drug therapy*
  • Leukemia, Myelomonocytic, Chronic / genetics
  • Leukemia, Myelomonocytic, Chronic / pathology
  • Mice
  • Mice, Transgenic
  • Myeloproliferative Disorders / drug therapy*
  • Myeloproliferative Disorders / genetics
  • Myeloproliferative Disorders / pathology
  • Oncogene Proteins, Fusion / genetics*
  • Piperazines / therapeutic use*
  • Pyrimidines / therapeutic use*
  • RUNX1 Translocation Partner 1 Protein
  • Spleen / metabolism
  • Spleen / pathology

Substances

  • AML1-ETO fusion protein, human
  • Antineoplastic Agents
  • Benzamides
  • Core Binding Factor Alpha 2 Subunit
  • DNA-Binding Proteins
  • HIP1 protein, human
  • Oncogene Proteins, Fusion
  • Piperazines
  • Pyrimidines
  • RUNX1 Translocation Partner 1 Protein
  • Imatinib Mesylate