In vitro and in vivo effects of a farnesyltransferase inhibitor on Nf1-deficient hematopoietic cells

Blood. 1999 Oct 1;94(7):2469-76.

Abstract

Oncogenic RAS alleles encode proteins that accumulate in the guanosine triphosphate (GTP)-bound state. Because post-translational processing of Ras by farnesyltransferase is essential for biologic function, inhibitors of this enzyme have been developed as rational cancer therapeutics. We have investigated farnesyltransferase inhibitor (FTI) L-744,832 in an in vivo murine model of myeloid leukemia that is associated with inactivation of the Nf1 tumor suppressor gene. Nf1 encodes a GTPase activating protein for Ras, and Nf1-deficient (Nf1-/-) hematopoietic cells show hyperactive Ras signaling through the mitogen-activated protein (MAP) kinase pathway. L-744,832 inhibited H-Ras prenylation in cell lines and in primary hematopoietic cells and abrogated the in vitro growth of myeloid progenitor colonies in response to granulocyte-macrophage colony-stimulating factor (GM-CSF). This FTI also partially blocked GM-CSF-induced MAP kinase activation, but did not reduce constitutively elevated levels of MAP kinase activity in primary Nf1-/- cells. Injection of a single dose of 40 or 80 mg/kg of L-744, 832 increased the amount of unprocessed H-Ras in bone marrow cells, but had no detectable effect on N-Ras. Adoptive transfer of Nf1-/- hematopoietic cells into irradiated mice induces a myeloproliferative disorder that did not respond to L-744,832 treatment. We speculate that the lack of efficacy in this model is due to the resistance of N-Ras and K-Ras processing to inhibition by this FTI.

Publication types

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

MeSH terms

  • Alkyl and Aryl Transferases / antagonists & inhibitors*
  • Animals
  • Antineoplastic Agents / pharmacology*
  • Cell Division / drug effects
  • Cells, Cultured
  • Colony-Forming Units Assay
  • Crosses, Genetic
  • Farnesyltranstransferase
  • Female
  • Genes, Tumor Suppressor*
  • Granulocyte-Macrophage Colony-Stimulating Factor / pharmacology
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / drug effects*
  • Hematopoietic Stem Cells / metabolism
  • Leukemia, Myeloid
  • Leukocyte Count / drug effects
  • Liver / cytology
  • Liver / embryology
  • Male
  • Methionine / analogs & derivatives*
  • Methionine / pharmacology
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred Strains
  • Mice, Knockout
  • Neurofibromin 1
  • Protein Prenylation
  • Proteins / genetics
  • Proteins / metabolism*
  • ras Proteins / metabolism

Substances

  • Antineoplastic Agents
  • L 744832
  • Neurofibromin 1
  • Proteins
  • Granulocyte-Macrophage Colony-Stimulating Factor
  • Methionine
  • Alkyl and Aryl Transferases
  • Farnesyltranstransferase
  • ras Proteins