Dual targeting of p53 and c-MYC selectively eliminates leukaemic stem cells

Nature. 2016 Jun 16;534(7607):341-6. doi: 10.1038/nature18288. Epub 2016 Jun 8.

Abstract

Chronic myeloid leukaemia (CML) arises after transformation of a haemopoietic stem cell (HSC) by the protein-tyrosine kinase BCR-ABL. Direct inhibition of BCR-ABL kinase has revolutionized disease management, but fails to eradicate leukaemic stem cells (LSCs), which maintain CML. LSCs are independent of BCR-ABL for survival, providing a rationale for identifying and targeting kinase-independent pathways. Here we show--using proteomics, transcriptomics and network analyses--that in human LSCs, aberrantly expressed proteins, in both imatinib-responder and non-responder patients, are modulated in concert with p53 (also known as TP53) and c-MYC regulation. Perturbation of both p53 and c-MYC, and not BCR-ABL itself, leads to synergistic cell kill, differentiation, and near elimination of transplantable human LSCs in mice, while sparing normal HSCs. This unbiased systems approach targeting connected nodes exemplifies a novel precision medicine strategy providing evidence that LSCs can be eradicated.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetamides / pharmacology
  • Acetamides / therapeutic use
  • Animals
  • Antigens, CD34 / metabolism
  • Azepines / pharmacology
  • Azepines / therapeutic use
  • Cell Death / drug effects
  • Cell Differentiation / drug effects
  • DNA-Binding Proteins / metabolism
  • Female
  • Fusion Proteins, bcr-abl / metabolism
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / drug effects
  • Hematopoietic Stem Cells / metabolism
  • Humans
  • Imatinib Mesylate / pharmacology
  • Imatinib Mesylate / therapeutic use
  • Imidazolines / pharmacology
  • Imidazolines / therapeutic use
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / genetics
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / metabolism*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / pathology*
  • Male
  • Mice
  • Neoplasm Proteins / metabolism
  • Neoplastic Stem Cells / drug effects
  • Neoplastic Stem Cells / metabolism*
  • Neoplastic Stem Cells / pathology*
  • Neoplastic Stem Cells / transplantation
  • Proteomics
  • Proto-Oncogene Proteins c-myc / antagonists & inhibitors*
  • Proto-Oncogene Proteins c-myc / deficiency
  • Proto-Oncogene Proteins c-myc / genetics
  • Proto-Oncogene Proteins c-myc / metabolism
  • Reproducibility of Results
  • Signal Transduction / drug effects
  • Transcriptome
  • Tumor Suppressor Protein p53 / antagonists & inhibitors*
  • Tumor Suppressor Protein p53 / deficiency
  • Tumor Suppressor Protein p53 / genetics
  • Tumor Suppressor Protein p53 / metabolism

Substances

  • Acetamides
  • Antigens, CD34
  • Azepines
  • CPI203
  • DNA-Binding Proteins
  • Imidazolines
  • MYC protein, human
  • Neoplasm Proteins
  • Proto-Oncogene Proteins c-myc
  • RG7112
  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • ZNF331 protein, human
  • Imatinib Mesylate
  • Fusion Proteins, bcr-abl