Characterization of Rare, Dormant, and Therapy-Resistant Cells in Acute Lymphoblastic Leukemia

Cancer Cell. 2016 Dec 12;30(6):849-862. doi: 10.1016/j.ccell.2016.11.002. Epub 2016 Dec 1.

Abstract

Tumor relapse is associated with dismal prognosis, but responsible biological principles remain incompletely understood. To isolate and characterize relapse-inducing cells, we used genetic engineering and proliferation-sensitive dyes in patient-derived xenografts of acute lymphoblastic leukemia (ALL). We identified a rare subpopulation that resembled relapse-inducing cells with combined properties of long-term dormancy, treatment resistance, and stemness. Single-cell and bulk expression profiling revealed their similarity to primary ALL cells isolated from pediatric and adult patients at minimal residual disease (MRD). Therapeutically adverse characteristics were reversible, as resistant, dormant cells became sensitive to treatment and started proliferating when dissociated from the in vivo environment. Our data suggest that ALL patients might profit from therapeutic strategies that release MRD cells from the niche.

Keywords: Cancer stem cells; RNA single-cell sequencing; acute lymphoblastic leukemia; dormant tumor cells; minimal residual disease (MRD); patient-derived xenograft (PDX) cells; primary patients' ALL MRD cells; treatment resistance.

MeSH terms

  • Adult
  • Animals
  • Antineoplastic Combined Chemotherapy Protocols / metabolism
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Cell Proliferation
  • Child
  • Disease-Free Survival
  • Drug Resistance, Neoplasm*
  • Gene Expression Profiling / methods*
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Mice
  • Neoplasm Recurrence, Local / genetics
  • Neoplasm Recurrence, Local / pathology*
  • Neoplasm Transplantation
  • Neoplasm, Residual / genetics
  • Neoplasm, Residual / pathology
  • Neoplastic Stem Cells / pathology*
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology*
  • Prognosis
  • Proto-Oncogene Proteins c-myc / genetics*
  • Sequence Analysis, RNA / methods*
  • Single-Cell Analysis
  • Tumor Cells, Cultured

Substances

  • Myc protein, mouse
  • Proto-Oncogene Proteins c-myc