Pre-clinical activity of combined LSD1 and mTORC1 inhibition in MLL-translocated acute myeloid leukaemia

Leukemia. 2020 May;34(5):1266-1277. doi: 10.1038/s41375-019-0659-6. Epub 2019 Nov 28.

Abstract

The histone demethylase lysine-specific demethylase 1 (LSD1 or KDM1A) has emerged as a candidate therapeutic target in acute myeloid leukaemia (AML); tranylcypromine-derivative inhibitors induce loss of clonogenic activity and promote differentiation, in particular in the MLL-translocated molecular subtype of AML. In AML, the use of drugs in combination often delivers superior clinical activity. To identify genes and cellular pathways that collaborate with LSD1 to maintain the leukaemic phenotype, and which could be targeted by combination therapies, we performed a genome-wide CRISPR-Cas9 dropout screen. We identified multiple components of the amino acid sensing arm of mTORC1 signalling-RRAGA, MLST8, WDR24 and LAMTOR2-as cellular sensitizers to LSD1 inhibition. Knockdown of mTORC1 components, or mTORC1 pharmacologic inhibition, in combination with LSD1 inhibition enhanced differentiation in both cell line and primary cell settings, in vitro and in vivo, and substantially reduced the frequency of clonogenic primary human AML cells in a modelled minimal residual disease setting. Synergistic upregulation of a set of transcription factor genes associated with terminal monocytic lineage differentiation was observed. Thus, dual mTORC1 and LSD1 inhibition represents a candidate combination approach for enhanced differentiation in MLL-translocated AML which could be evaluated in early phase clinical trials.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antidepressive Agents / pharmacology
  • Antineoplastic Agents / pharmacology
  • Apoptosis
  • Cell Proliferation
  • Drug Therapy, Combination
  • Everolimus / pharmacology*
  • Female
  • Gene Expression Regulation, Leukemic
  • Histone Demethylases / antagonists & inhibitors*
  • Histone-Lysine N-Methyltransferase / genetics*
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / metabolism
  • Leukemia, Myeloid, Acute / pathology
  • Mechanistic Target of Rapamycin Complex 1 / antagonists & inhibitors*
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Myeloid-Lymphoid Leukemia Protein / genetics*
  • Translocation, Genetic*
  • Tranylcypromine / pharmacology*
  • Tumor Cells, Cultured
  • Xenograft Model Antitumor Assays

Substances

  • Antidepressive Agents
  • Antineoplastic Agents
  • KMT2A protein, human
  • Myeloid-Lymphoid Leukemia Protein
  • Tranylcypromine
  • Everolimus
  • Histone Demethylases
  • KDM1A protein, human
  • Histone-Lysine N-Methyltransferase
  • Mechanistic Target of Rapamycin Complex 1