Center for Translational Medicine, Jane and Leonard Korman Lung Center, and.
Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, Pennsylvania; and.
Am J Respir Cell Mol Biol. 2023 Jan;68(1):23-38. doi: 10.1165/rcmb.2022-0110OC.
ERK1/2 (extracellular signal-regulated kinases 1 and 2) regulate the activity of various transcription factors that contribute to asthma pathogenesis. Although an attractive drug target, broadly inhibiting ERK1/2 is challenging because of unwanted cellular toxicities. We have identified small molecule inhibitors with a benzenesulfonate scaffold that selectively inhibit ERK1/2-mediated activation of AP-1 (activator protein-1). Herein, we describe the findings of targeting ERK1/2-mediated substrate-specific signaling with the small molecule inhibitor SF-3-030 in a murine model of house dust mite (HDM)-induced asthma. In 8- to 10-week-old BALB/c mice, allergic asthma was established by repeated intranasal HDM (25 μg/mouse) instillation for 3 weeks (5 days/week). A subgroup of mice was prophylactically dosed with 10 mg/kg SF-3-030/DMSO intranasally 30 minutes before the HDM challenge. Following the dosing schedule, mice were evaluated for alterations in airway mechanics, inflammation, and markers of airway remodeling. SF-3-030 treatment significantly attenuated HDM-induced elevation of distinct inflammatory cell types and cytokine concentrations in BAL and IgE concentrations in the lungs. Histopathological analysis of lung tissue sections revealed diminished HDM-induced pleocellular peribronchial inflammation, mucus cell metaplasia, collagen accumulation, thickening of airway smooth muscle mass, and expression of markers of cell proliferation (Ki-67 and cyclin D1) in mice treated with SF-3-030. Furthermore, SF-3-030 treatment attenuated HDM-induced airway hyperresponsiveness in mice. Finally, mechanistic studies using transcriptome and proteome analyses suggest inhibition of HDM-induced genes involved in inflammation, cell proliferation, and tissue remodeling by SF-3-030. These preclinical findings demonstrate that function-selective inhibition of ERK1/2 signaling mitigates multiple features of asthma in a murine model.
ERK1/2(细胞外信号调节激酶 1 和 2)调节各种转录因子的活性,这些转录因子有助于哮喘发病机制。尽管 ERK1/2 是一个有吸引力的药物靶点,但由于细胞毒性的原因,广泛抑制 ERK1/2 具有挑战性。我们已经确定了具有苯磺酸盐支架的小分子抑制剂,该抑制剂选择性抑制 ERK1/2 介导的 AP-1(激活蛋白-1)的激活。在此,我们描述了用小分子抑制剂 SF-3-030 靶向 ERK1/2 介导的底物特异性信号在屋尘螨(HDM)诱导的哮喘小鼠模型中的发现。在 8-10 周龄的 BALB/c 小鼠中,通过重复鼻腔内给予 25 μg/只的 HDM(每周 5 天,共 3 周)来建立过敏性哮喘。一组小鼠在 HDM 攻击前 30 分钟用 10 mg/kg SF-3-030/DMSO 经鼻腔预防性给药。按照给药方案,评估小鼠气道力学、炎症和气道重塑标志物的变化。SF-3-030 治疗显著减轻了 HDM 诱导的 BAL 中不同炎症细胞类型和细胞因子浓度以及肺部 IgE 浓度的升高。肺组织切片的组织病理学分析显示,SF-3-030 治疗减轻了 HDM 诱导的多细胞性支气管周围炎症、粘液细胞化生、胶原积累、气道平滑肌质量增厚和细胞增殖标志物(Ki-67 和 cyclin D1)在小鼠中的表达。此外,SF-3-030 治疗减轻了 HDM 诱导的小鼠气道高反应性。最后,使用转录组和蛋白质组分析的机制研究表明,SF-3-030 抑制了 HDM 诱导的炎症、细胞增殖和组织重塑相关基因的表达。这些临床前研究结果表明,ERK1/2 信号的功能选择性抑制减轻了哮喘小鼠模型中的多种哮喘特征。