Hubei Key Laboratory of Cell Homeostasis, College of Life Sciences, Wuhan University, Wuhan 430072, China.
Hubei Provincial Key Laboratory of Developmentally Originated Disease, Frontier Science Center for Immunology and Metabolism, Key Laboratory of Combinatorial Biosynthesis and Drug Discovery (Wuhan University), Ministry of Education, Wuhan University School of Pharmaceutical Sciences, Wuhan 430071, China.
Bioorg Med Chem. 2021 Jun 15;40:116185. doi: 10.1016/j.bmc.2021.116185. Epub 2021 May 1.
Hormone therapy targeting estrogen receptors is widely used clinically for the treatment of breast cancer, such as tamoxifen, but most of them are partial agonists, which can cause serious side effects after long-term use. The use of selective estrogen receptor down-regulators (SERDs) may be an effective alternative to breast cancer therapy by directly degrading ERα protein to shut down ERα signaling. However, the solely clinically used SERD fulvestrant, is low orally bioavailable and requires intravenous injection, which severely limits its clinical application. On the other hand, double- or multi-target conjugates, which are able to synergize antitumor activity by different pathways, thus may enhance therapeutic effect in comparison with single targeted therapy. In this study, we designed and synthesized a series of novel dual-functional conjugates targeting both ERα degradation and histone deacetylase inhibiton by combining a privileged SERD skeleton 7-oxabicyclo[2.2.1]heptane sulfonamide (OBHSA) with a histone deacetylase inhibitor side chain. We found that substituents on both the sulfonamide nitrogen and phenyl group of OBHSA unit had significant effect on biological activities. Among them, conjugate 16i with N-methyl and naphthyl groups exhibited potent antiproliferative activity against MCF-7 cells, and excellent ERα degradation activity and HDACs inhibitory ability. A further molecular docking study indicated the interaction patterns of these conjugates with ERα, which may provide guidance to design novel SERDs or PROTAC-like SERDs for breast cancer therapy.
针对雌激素受体的激素疗法在临床上被广泛用于治疗乳腺癌,例如他莫昔芬,但大多数都是部分激动剂,长期使用会导致严重的副作用。选择性雌激素受体降解剂 (SERD) 的使用可能是乳腺癌治疗的有效替代方法,它可以通过直接降解 ERα 蛋白来关闭 ERα 信号。然而,临床上唯一使用的 SERD 氟维司群,口服生物利用度低,需要静脉注射,这严重限制了其临床应用。另一方面,双靶或多靶缀合物能够通过不同途径协同抗肿瘤活性,因此与单靶治疗相比,可能会增强治疗效果。在这项研究中,我们设计并合成了一系列新型的双重功能缀合物,通过将具有特权的 SERD 骨架 7-氧杂双环[2.2.1]庚烷磺酰胺 (OBHSA) 与组蛋白去乙酰化酶抑制剂侧链结合,靶向 ERα 降解和组蛋白去乙酰化酶抑制。我们发现,OBHSA 单元上的磺酰胺氮和苯环上的取代基对生物活性有显著影响。其中,带有 N-甲基和萘基的缀合物 16i 对 MCF-7 细胞表现出很强的增殖抑制活性,以及优异的 ERα 降解活性和 HDAC 抑制能力。进一步的分子对接研究表明了这些缀合物与 ERα 的相互作用模式,这可能为设计用于乳腺癌治疗的新型 SERD 或 PROTAC 样 SERD 提供指导。