Discovery of Small-Molecule Antagonists of the PWWP Domain of NSD2

J Med Chem. 2021 Feb 11;64(3):1584-1592. doi: 10.1021/acs.jmedchem.0c01768. Epub 2021 Feb 1.

Abstract

Increased activity of the lysine methyltransferase NSD2 driven by translocation and activating mutations is associated with multiple myeloma and acute lymphoblastic leukemia, but no NSD2-targeting chemical probe has been reported to date. Here, we present the first antagonists that block the protein-protein interaction between the N-terminal PWWP domain of NSD2 and H3K36me2. Using virtual screening and experimental validation, we identified the small-molecule antagonist 3f, which binds to the NSD2-PWWP1 domain with a Kd of 3.4 μM and abrogates histone H3K36me2 binding to the PWWP1 domain in cells. This study establishes an alternative approach to targeting NSD2 and provides a small-molecule antagonist that can be further optimized into a chemical probe to better understand the cellular function of this protein.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacology*
  • Computer Simulation
  • Crystallography, X-Ray
  • Drug Discovery / methods
  • Drug Evaluation, Preclinical
  • Drug Screening Assays, Antitumor
  • Histone-Lysine N-Methyltransferase / antagonists & inhibitors*
  • Histone-Lysine N-Methyltransferase / drug effects
  • Humans
  • Ligands
  • Models, Molecular
  • Molecular Docking Simulation
  • Protein Domains
  • Repressor Proteins / antagonists & inhibitors*
  • Repressor Proteins / drug effects
  • Small Molecule Libraries
  • Structure-Activity Relationship

Substances

  • Antineoplastic Agents
  • Ligands
  • Repressor Proteins
  • Small Molecule Libraries
  • Histone-Lysine N-Methyltransferase
  • NSD2 protein, human