Identification of Novel Potent NSD2-PWWP1 Ligands Using Structure-Based Design and Computational Approaches

J Med Chem. 2024 Jun 13;67(11):8962-8987. doi: 10.1021/acs.jmedchem.4c00215. Epub 2024 May 15.

Abstract

Dysregulation of histone methyl transferase nuclear receptor-binding SET domain 2 (NSD2) has been implicated in several hematological and solid malignancies. NSD2 is a large multidomain protein that carries histone writing and histone reading functions. To date, identifying inhibitors of the enzymatic activity of NSD2 has proven challenging in terms of potency and SET domain selectivity. Inhibition of the NSD2-PWWP1 domain using small molecules has been considered as an alternative approach to reduce NSD2-unregulated activity. In this article, we present novel computational chemistry approaches, encompassing free energy perturbation coupled to machine learning (FEP/ML) models as well as virtual screening (VS) activities, to identify high-affinity NSD2 PWWP1 binders. Through these activities, we have identified the most potent NSD2-PWWP1 binder reported so far in the literature: compound 34 (pIC50 = 8.2). The compounds identified herein represent useful tools for studying the role of PWWP1 domains for inhibition of human NSD2.

MeSH terms

  • Drug Design*
  • Histone-Lysine N-Methyltransferase* / antagonists & inhibitors
  • Histone-Lysine N-Methyltransferase* / chemistry
  • Histone-Lysine N-Methyltransferase* / metabolism
  • Humans
  • Ligands
  • Machine Learning
  • Models, Molecular
  • Protein Domains
  • Repressor Proteins / antagonists & inhibitors
  • Repressor Proteins / chemistry
  • Repressor Proteins / metabolism
  • Structure-Activity Relationship

Substances

  • Histone-Lysine N-Methyltransferase
  • Ligands
  • NSD2 protein, human
  • Repressor Proteins