Growth arrest of BCR-ABL positive cells with a sequence-specific polyamide-chlorambucil conjugate

PLoS One. 2008;3(10):e3593. doi: 10.1371/journal.pone.0003593. Epub 2008 Oct 31.

Abstract

Chronic myeloid leukemia (CML) is characterized by the presence of a constitutively active Abl kinase, which is the product of a chimeric BCR-ABL gene, caused by the genetic translocation known as the Philadelphia chromosome. Imatinib, a selective inhibitor of the Bcr-Abl tyrosine kinase, has significantly improved the clinical outcome of patients with CML. However, subsets of patients lose their response to treatment through the emergence of imatinib-resistant cells, and imatinib treatment is less durable for patients with late stage CML. Although alternative Bcr-Abl tyrosine kinase inhibitors have been developed to overcome drug resistance, a cocktail therapy of different kinase inhibitors and additional chemotherapeutics may be needed for complete remission of CML in some cases. Chlorambucil has been used for treatment of B cell chronic lymphocytic leukemia, non-Hodgkin's and Hodgkin's disease. Here we report that a DNA sequence-specific pyrrole-imidazole polyamide-chlorambucil conjugate, 1R-Chl, causes growth arrest of cells harboring both unmutated BCR-ABL and three imatinib resistant strains. 1R-Chl also displays selective toxicities against activated lymphocytes and a high dose tolerance in a murine model.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents, Alkylating / administration & dosage
  • Antineoplastic Agents, Alkylating / pharmacology
  • Base Sequence / drug effects
  • Benzamides
  • Blood Cells / metabolism
  • Blood Cells / pathology
  • Bone Marrow Cells / metabolism
  • Bone Marrow Cells / physiology
  • Cell Proliferation / drug effects*
  • Cells, Cultured
  • Chlorambucil / administration & dosage
  • Chlorambucil / chemistry
  • Chlorambucil / pharmacology*
  • Drug Evaluation, Preclinical
  • Fusion Proteins, bcr-abl / genetics*
  • Fusion Proteins, bcr-abl / metabolism
  • Humans
  • Imatinib Mesylate
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / pathology
  • Mice
  • Models, Biological
  • Nylons / chemistry
  • Nylons / pharmacology*
  • Piperazines / administration & dosage
  • Piperazines / pharmacology
  • Pyrimidines / administration & dosage
  • Pyrimidines / pharmacology
  • Substrate Specificity / drug effects
  • Transduction, Genetic

Substances

  • Antineoplastic Agents, Alkylating
  • Benzamides
  • Nylons
  • Piperazines
  • Pyrimidines
  • Chlorambucil
  • Imatinib Mesylate
  • Fusion Proteins, bcr-abl