A potent and selective ENL degrader suppresses oncogenic gene expression and leukemia progression

Sci Adv. 2024 Aug 30;10(35):eado1432. doi: 10.1126/sciadv.ado1432. Epub 2024 Aug 28.

Abstract

The histone acylation reader eleven-nineteen leukemia (ENL) plays a pivotal role in sustaining oncogenesis in acute leukemias, particularly in mixed-lineage leukemia-rearranged (MLL-r) leukemia. ENL relies on its reader domain to recognize histone lysine acylation promoting oncogenic gene expression and leukemia progression. Here, we report the development of MS41, a highly potent and selective von Hippel-Lindau-recruiting ENL degrader that effectively inhibits the growth of ENL-dependent leukemia cells. MS41-induced ENL degradation reduces the chromatin occupancy of ENL-associated transcription elongation machinery, resulting in the suppression of key oncogenic gene expression programs and the activation of differentiation genes. MS41 is well-tolerated in vivo and substantially suppresses leukemia progression in a xenograft mouse model of MLL-r leukemia. Notably, MS41 also induces the degradation of mutant ENL proteins identified in Wilms' tumors. Our findings emphasize the therapeutic potential of pharmacological ENL degradation for treating ENL-dependent cancers, making MS41 not only a valuable chemical probe but also potential anticancer therapeutic for further development.

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Disease Progression*
  • Gene Expression Regulation, Leukemic / drug effects
  • Humans
  • Leukemia* / drug therapy
  • Leukemia* / genetics
  • Leukemia* / metabolism
  • Leukemia* / pathology
  • Mice
  • Proteolysis / drug effects
  • Transcriptional Elongation Factors / genetics
  • Transcriptional Elongation Factors / metabolism
  • Von Hippel-Lindau Tumor Suppressor Protein / genetics
  • Von Hippel-Lindau Tumor Suppressor Protein / metabolism
  • Xenograft Model Antitumor Assays

Substances

  • ELL protein, human
  • Transcriptional Elongation Factors
  • Von Hippel-Lindau Tumor Suppressor Protein