Discovery of Potent, Selective, and Orally Bioavailable Estrogen-Related Receptor-γ Inverse Agonists To Restore the Sodium Iodide Symporter Function in Anaplastic Thyroid Cancer

J Med Chem. 2019 Feb 28;62(4):1837-1858. doi: 10.1021/acs.jmedchem.8b01296. Epub 2019 Feb 4.

Abstract

An inverse agonist of estrogen-related receptor-γ (ERRγ), an orphan nuclear receptor encoded by E srrg, enhances sodium iodide symporter-mediated radioiodine uptake in anaplastic thyroid cancer (ATC) cells, thereby facilitating responsiveness to radioiodine therapy in vitro. We synthesized potent, selective, and orally bioavailable ERRγ-inverse agonists and evaluated their activity by analyzing in vitro pharmacology and absorption, distribution, metabolism, excretion, and toxicity profiles. X-ray crystallographic analysis of the ligand and ERRγ complex showed that 35 completely binds to the target protein (PDB 6A6K ). Our results showed improved radioiodine avidity in ATC cells through compound 35-mediated upregulation of iodide-handling genes, leading to enhanced responsiveness to radioiodine therapy in vitro. Importantly, in vivo 124I-positron emission tomography/computed tomography imaging revealed that 35 increases radioiodine avidity in CAL62 tumors. Collectively, these results demonstrated that 35 can be developed as a promising treatment for ERRγ-related cancer in the future.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / therapeutic use
  • Cell Line, Tumor
  • Drug Discovery
  • Drug Inverse Agonism
  • Estrogens / agonists
  • Estrogens / chemical synthesis
  • Estrogens / pharmacokinetics
  • Estrogens / therapeutic use
  • Female
  • Gene Expression / drug effects
  • Humans
  • Iodine Radioisotopes / metabolism
  • Mice, Inbred BALB C
  • Molecular Structure
  • Receptors, Estrogen / metabolism*
  • Structure-Activity Relationship
  • Symporters / metabolism*
  • Tamoxifen / agonists
  • Tamoxifen / analogs & derivatives*
  • Tamoxifen / pharmacokinetics
  • Tamoxifen / therapeutic use*
  • Thyroid Carcinoma, Anaplastic / drug therapy*
  • Thyroid Carcinoma, Anaplastic / metabolism
  • Thyroid Neoplasms / drug therapy*
  • Thyroid Neoplasms / metabolism

Substances

  • Antineoplastic Agents
  • ESRRG protein, human
  • Estrogens
  • Iodine Radioisotopes
  • Iodine-124
  • Iodine-131
  • Receptors, Estrogen
  • Symporters
  • Tamoxifen
  • sodium-iodide symporter