Structure-Based Design of a Potent and Selective YTHDC1 Ligand

J Med Chem. 2024 Jun 13;67(11):9516-9535. doi: 10.1021/acs.jmedchem.4c00599. Epub 2024 May 24.

Abstract

N6-Adenosine methylation (m6A) is a prevalent post-transcriptional modification of mRNA, with YTHDC1 being the reader protein responsible for recognizing this modification in the cell nucleus. Here, we present a protein structure-based medicinal chemistry campaign that resulted in the YTHDC1 inhibitor 40, which shows an equilibrium dissociation constant (Kd) of 49 nM. The crystal structure of the complex (1.6 Å resolution) validated the design. Compound 40 is selective against the cytoplasmic m6A-RNA readers YTHDF1-3 and YTHDC2 and shows antiproliferative activity against the acute myeloid leukemia (AML) cell lines THP-1, MOLM-13, and NOMO-1. For the series of compounds that culminated into ligand 40, the good correlation between the affinity in the biochemical assay and antiproliferative activity in the THP-1 cell line provides evidence of YTHDC1 target engagement in the cell. The binding to YTHDC1 in the cell is further supported by the cellular thermal shift assay. Thus, ligand 40 is a tool compound for studying the role of YTHDC1 in AML.

MeSH terms

  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Crystallography, X-Ray
  • Drug Design*
  • Heterocyclic Compounds, 2-Ring / chemistry
  • Heterocyclic Compounds, 2-Ring / pharmacology
  • Humans
  • Ligands
  • Models, Molecular
  • Nerve Tissue Proteins
  • RNA Splicing Factors / chemistry
  • RNA Splicing Factors / metabolism
  • Structure-Activity Relationship

Substances

  • Antineoplastic Agents
  • Ligands
  • Nerve Tissue Proteins
  • RNA Splicing Factors
  • YTHDC1 protein, human
  • Heterocyclic Compounds, 2-Ring