Preclinical evaluation of a regimen combining chidamide and ABT-199 in acute myeloid leukemia

Cell Death Dis. 2020 Sep 18;11(9):778. doi: 10.1038/s41419-020-02972-2.

Abstract

Acute myeloid leukemia (AML) is a heterogeneous myeloid neoplasm with poor clinical outcome, despite the great progress in treatment in recent years. The selective Bcl-2 inhibitor venetoclax (ABT-199) in combination therapy has been approved for the treatment of newly diagnosed AML patients who are ineligible for intensive chemotherapy, but resistance can be acquired through the upregulation of alternative antiapoptotic proteins. Here, we reported that a newly emerged histone deacetylase inhibitor, chidamide (CS055), at low-cytotoxicity dose enhanced the anti-AML activity of ABT-199, while sparing normal hematopoietic progenitor cells. Moreover, we also found that chidamide showed a superior resensitization effect than romidepsin in potentiation of ABT-199 lethality. Inhibition of multiple HDACs rather than some single component might be required. The combination therapy was also effective in primary AML blasts and stem/progenitor cells regardless of disease status and genetic aberrance, as well as in a patient-derived xenograft model carrying FLT3-ITD mutation. Mechanistically, CS055 promoted leukemia suppression through DNA double-strand break and altered unbalance of anti- and pro-apoptotic proteins (e.g., Mcl-1 and Bcl-xL downregulation, and Bim upregulation). Taken together, these results show the high therapeutic potential of ABT-199/CS055 combination in AML treatment, representing a potent and alternative salvage therapy for the treatment of relapsed and refractory patients with AML.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aminopyridines / pharmacology*
  • Antineoplastic Agents / pharmacology
  • Apoptosis Regulatory Proteins / drug effects
  • Apoptosis Regulatory Proteins / metabolism
  • Benzamides / pharmacology*
  • Biphenyl Compounds / pharmacology
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology*
  • Cell Proliferation / drug effects*
  • Humans
  • Leukemia, Myeloid, Acute / chemically induced
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / metabolism
  • Sulfonamides / pharmacology*

Substances

  • Aminopyridines
  • Antineoplastic Agents
  • Apoptosis Regulatory Proteins
  • Benzamides
  • Biphenyl Compounds
  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • N-(2-amino-5-fluorobenzyl)-4-(N-(pyridine-3-acrylyl)aminomethyl)benzamide
  • venetoclax