Design, Synthesis, and Biological Evaluation of 3,4-Dihydroisoquinolin-1(2H)-one Derivatives as Protein Arginine Methyltransferase 5 Inhibitors for the Treatment of Non-Hodgkin's Lymphoma

J Med Chem. 2025 Jan 9;68(1):108-134. doi: 10.1021/acs.jmedchem.4c01548. Epub 2024 Dec 26.

Abstract

Through catalyzing the transfer of methyl groups onto the guanidinium of arginine, protein arginine methyltransferase 5 (PRMT5) was essential to the cell growth of cancer cells. By utilizing a scaffold hopping strategy, a novel series of 3,4-dihydroisoquinolin-1(2H)-one derivatives were designed and synthesized. Through a systematic SAR study, D3 demonstrated excellent PRMT5 inhibitory activity, potent antiproliferative activity against Z-138, favorable pharmacokinetic profiles, and low hERG toxicity. Molecular docking, molecular dynamic (MD) simulation, and surface plasmon resonance (SPR) study indicated that D3 was tightly interacted with PRMT5. Meanwhile, D3 exhibited high selectivity against PRMT5, which could inhibit the growth of various cancer cells, induce apoptosis, and arrest the cell cycle in the G0/G1 phase. Additionally, D3 possessed excellent antitumor efficacy in Z-138 xenograft models, low toxicity in vivo, and acceptable drug metabolism and pharmacokinetics (DMPK) profiles in vitro. Therefore, D3 can be developed as a promising candidate for the treatment of non-Hodgkin's lymphoma (NHL).

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacokinetics
  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Apoptosis / drug effects
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Drug Design*
  • Enzyme Inhibitors / chemical synthesis
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology
  • Enzyme Inhibitors / therapeutic use
  • Humans
  • Isoquinolines* / chemical synthesis
  • Isoquinolines* / chemistry
  • Isoquinolines* / pharmacokinetics
  • Isoquinolines* / pharmacology
  • Isoquinolines* / therapeutic use
  • Lymphoma, Non-Hodgkin* / drug therapy
  • Lymphoma, Non-Hodgkin* / pathology
  • Male
  • Mice
  • Mice, Nude
  • Molecular Docking Simulation
  • Protein-Arginine N-Methyltransferases* / antagonists & inhibitors
  • Protein-Arginine N-Methyltransferases* / metabolism
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Protein-Arginine N-Methyltransferases
  • Isoquinolines
  • Antineoplastic Agents
  • PRMT5 protein, human
  • Enzyme Inhibitors