Synthesis of 5,9- and 5,8-Diaminoalkoxy Substituted Benzophenanthridinone Analogues as Tyrosyl-DNA Phosphodiesterase 1 Inhibitors and Their Radiosensitizing Activity

J Med Chem. 2025 May 8;68(9):9323-9340. doi: 10.1021/acs.jmedchem.4c02951. Epub 2025 Apr 29.

Abstract

Tyrosyl-DNA phosphodiesterase 1 (TDP1) is a potential target for cancer chemotherapy and radiotherapy. There are a few reports on TDP1 inhibitors used in chemotherapy, but no report on their use in radiotherapy. Herein, we designed and synthesized a series of titled analogues. Twelve analogues showed high TDP1 inhibitory activity. Among them, 18 (IC50 = 6.9 μM) showed strong radiosensitization in colorectal cancer cells, and could suppress tumor growth in the HCT116 xenograft animal model combined with X-ray radiation, and exhibited low acute toxicity with good pharmacokinetic (PK) parameters, implying that 18 is worth further clinical research. Further studies indicated that 18 could target cellular TDP1 and suppress NHEJ repair activity for radiation-induced DNA damage, resulting in cancer cell death. Additionally, 18 could also increase the expression of PIG3, resulting in an enhancement of radiation-induced cellular ROS and mitochondrial dysfunction. Our studies provide a novel cancer treatment strategy combining TDP1 inhibitors and radiotherapy.

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / pharmacology
  • Cell Line, Tumor
  • HCT116 Cells
  • Humans
  • Mice
  • Mice, Nude
  • Phosphodiesterase Inhibitors* / chemical synthesis
  • Phosphodiesterase Inhibitors* / chemistry
  • Phosphodiesterase Inhibitors* / pharmacology
  • Phosphoric Diester Hydrolases* / metabolism
  • Radiation-Sensitizing Agents* / chemical synthesis
  • Radiation-Sensitizing Agents* / chemistry
  • Radiation-Sensitizing Agents* / pharmacology
  • Radiation-Sensitizing Agents* / therapeutic use
  • Reactive Oxygen Species / metabolism
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Phosphoric Diester Hydrolases
  • Phosphodiesterase Inhibitors
  • Radiation-Sensitizing Agents
  • TDP1 protein, human
  • Antineoplastic Agents
  • Reactive Oxygen Species