Variable behavior of iPSCs derived from CML patients for response to TKI and hematopoietic differentiation

PLoS One. 2013 Aug 23;8(8):e71596. doi: 10.1371/journal.pone.0071596. eCollection 2013.

Abstract

Chronic myeloid leukemia disease (CML) found effective therapy by treating patients with tyrosine kinase inhibitors (TKI), which suppress the BCR-ABL1 oncogene activity. However, the majority of patients achieving remission with TKI still have molecular evidences of disease persistence. Various mechanisms have been proposed to explain the disease persistence and recurrence. One of the hypotheses is that the primitive leukemic stem cells (LSCs) can survive in the presence of TKI. Understanding the mechanisms leading to TKI resistance of the LSCs in CML is a critical issue but is limited by availability of cells from patients. We generated induced pluripotent stem cells (iPSCs) derived from CD34⁺ blood cells isolated from CML patients (CML-iPSCs) as a model for studying LSCs survival in the presence of TKI and the mechanisms supporting TKI resistance. Interestingly, CML-iPSCs resisted to TKI treatment and their survival did not depend on BCR-ABL1, as for primitive LSCs. Induction of hematopoietic differentiation of CML-iPSC clones was reduced compared to normal clones. Hematopoietic progenitors obtained from iPSCs partially recovered TKI sensitivity. Notably, different CML-iPSCs obtained from the same CML patients were heterogeneous, in terms of BCR-ABL1 level and proliferation. Thus, several clones of CML-iPSCs are a powerful model to decipher all the mechanisms leading to LSC survival following TKI therapy and are a promising tool for testing new therapeutic agents.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Resistance, Neoplasm*
  • Fusion Proteins, bcr-abl / antagonists & inhibitors*
  • Hematopoiesis / drug effects*
  • Humans
  • Induced Pluripotent Stem Cells / cytology
  • Induced Pluripotent Stem Cells / drug effects*
  • Induced Pluripotent Stem Cells / pathology*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / pathology
  • Protein Kinase Inhibitors / pharmacology*
  • Tumor Cells, Cultured

Substances

  • BCR-ABL1 fusion protein, human
  • Protein Kinase Inhibitors
  • Fusion Proteins, bcr-abl

Grants and funding

This work was supported by grants from Fondation de France and from Novartis pharmaceutical company. The authors thank Novartis for providing them with imatinib. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.