Discovery of new LXRβ agonists as glioblastoma inhibitors

  • Eur J Med Chem. 2020 May 15;194:112240. doi: 10.1016/j.ejmech.2020.112240.
Hao Chen  1 Ziyang Chen  1 Zizhen Zhang  1 Yali Li  1 Shushu Zhang  1 Fuqiang Jiang  1 Junkang Wei  1 Peng Ding  1 Huihao Zhou  2 Qiong Gu  3 Jun Xu  4
Affiliations
  • 1. Guangdong Provincial Key Laboratory of New Drug Design and Evaluation, Research Center for Drug Discovery at School of Pharmaceutical Sciences, Sun Yat-Sen University, Guangzhou, 510006, China.
  • 2. Guangdong Provincial Key Laboratory of New Drug Design and Evaluation, Research Center for Drug Discovery at School of Pharmaceutical Sciences, Sun Yat-Sen University, Guangzhou, 510006, China. Electronic address: [email protected].
  • 3. Guangdong Provincial Key Laboratory of New Drug Design and Evaluation, Research Center for Drug Discovery at School of Pharmaceutical Sciences, Sun Yat-Sen University, Guangzhou, 510006, China. Electronic address: [email protected].
  • 4. Guangdong Provincial Key Laboratory of New Drug Design and Evaluation, Research Center for Drug Discovery at School of Pharmaceutical Sciences, Sun Yat-Sen University, Guangzhou, 510006, China. Electronic address: [email protected].
Abstract

Discovery and optimization of selective liver X receptor β (LXRβ) agonists are challenging due to the high homology of LXRα and LXRβ in the ligand-binding domain. There is only one different residue (Val versus Ile) at the ligand-binding pocket of LXRs. With machine learning methods, we identified pan LXR agonists with a novel scaffold (spiro[pyrrolidine-3,3'-oxindole]). Then, we figured out the mechanism of LXR isoform selectivity from co-crystal structures. Based on the mechanism and the new scaffold, LXRβ selective agonists were designed and synthesized. This led to the discovery of LXRβ agonists 4-7rr, 4-13 and 4-13rr with IC50 values ranging from 1.78 to 6.36 μM against glioblastoma in vitro. Treatment with 50 mg/kg/day of 4-13 for 15 days significantly reduced tumor growth using an in vivo xenograft glioblastoma model.

Keywords
Glioblastoma inhibitor; Nuclear receptor; Spiro[pyrrolidine-3,3′-oxindole]; Structure-based drug design.
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