Menin is necessary for long term maintenance of meningioma-1 driven leukemia

Leukemia. 2021 May;35(5):1405-1417. doi: 10.1038/s41375-021-01146-z. Epub 2021 Feb 4.

Abstract

Translocations of Meningioma-1 (MN1) occur in a subset of acute myeloid leukemias (AML) and result in high expression of MN1, either as a full-length protein, or as a fusion protein that includes most of the N-terminus of MN1. High levels of MN1 correlate with poor prognosis. When overexpressed in murine hematopoietic progenitors, MN1 causes an aggressive AML characterized by an aberrant myeloid precursor-like gene expression program that shares features of KMT2A-rearranged (KMT2A-r) leukemia, including high levels of Hoxa and Meis1 gene expression. Compounds that target a critical KMT2A-Menin interaction have proven effective in KMT2A-r leukemia. Here, we demonstrate that Menin (Men1) is also critical for the self-renewal of MN1-driven AML through the maintenance of a distinct gene expression program. Genetic inactivation of Men1 led to a decrease in the number of functional leukemia-initiating cells. Pharmacologic inhibition of the KMT2A-Menin interaction decreased colony-forming activity, induced differentiation programs in MN1-driven murine leukemia and decreased leukemic burden in a human AML xenograft carrying an MN1-ETV6 translocation. Collectively, these results nominate Menin inhibition as a promising therapeutic strategy in MN1-driven leukemia.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Gene Expression Regulation, Leukemic / genetics
  • HEK293 Cells
  • Histone-Lysine N-Methyltransferase / genetics
  • Humans
  • Leukemia, Myeloid, Acute / genetics*
  • Mice
  • Mice, Knockout
  • Proto-Oncogene Proteins / genetics*
  • Trans-Activators / genetics*
  • Tumor Suppressor Proteins / genetics*

Substances

  • Men1 protein, mouse
  • Mn1 protein, mouse
  • Proto-Oncogene Proteins
  • Trans-Activators
  • Tumor Suppressor Proteins
  • Histone-Lysine N-Methyltransferase