Targeting PFKFB3 sensitizes chronic myelogenous leukemia cells to tyrosine kinase inhibitor

Oncogene. 2018 May;37(21):2837-2849. doi: 10.1038/s41388-018-0157-8. Epub 2018 Mar 7.

Abstract

Resistance to the BCR-ABL tyrosine kinase inhibitor (TKI) remains a challenge for curing the disease in chronic myeloid leukemia (CML) patients as leukemia cells may survive through BCR-ABL kinase activity-independent signal pathways. To gain insight into BCR-ABL kinase activity-independent mechanisms, we performed an initial bioinformatics screen and followed by a quantitative PCR screen of genes that were elevated in CML samples. A total of 33 candidate genes were identified to be highly expressed in TKIs resistant patients. Among those genes, 6-phosphofructo-2-kinase/fructose-2,6-biphosphatase 3 (PFKFB3), controlling the limiting step of glycolysis, was found to be strongly associated with TKIs resistance. PFKFB3 knockdown or pharmacological inhibition of its kinase activity markedly enhanced the sensitivity of CML cells to TKIs. Furthermore, pharmacological inhibition of PFKFB3 inhibited CML cells growth and significantly prolonged the survival of both allograft and xenograft CML mice. ChIP-seq data analysis combined with subsequent knockdown experiment showed that the Ets transcription factor PU.1 regulated the elevated expression of PFKFB3 in TKIs-resistant CML cells. Therefore, our results showed that targeting PFKFB3 sensitizes CML cells to TKIs and PFKFB3 may be a potential BCR-ABL kinase activity-independent mechanism in CML.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antigens, Surface / drug effects
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Drug Resistance, Neoplasm* / drug effects
  • Drug Synergism
  • Enzyme Inhibitors / administration & dosage*
  • Enzyme Inhibitors / pharmacology
  • Gene Expression Profiling
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Imatinib Mesylate / administration & dosage
  • Imatinib Mesylate / pharmacology
  • Jurkat Cells
  • K562 Cells
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / genetics*
  • Mice
  • Phosphofructokinase-2 / genetics*
  • Protein Kinase Inhibitors / administration & dosage*
  • Protein Kinase Inhibitors / pharmacology
  • Proto-Oncogene Proteins / genetics*
  • Trans-Activators / genetics*
  • Up-Regulation
  • Xenograft Model Antitumor Assays

Substances

  • Antigens, Surface
  • Enzyme Inhibitors
  • Protein Kinase Inhibitors
  • Proto-Oncogene Proteins
  • Trans-Activators
  • proto-oncogene protein Spi-1
  • Imatinib Mesylate
  • PFKFB3 protein, human
  • Phosphofructokinase-2