Jefferson Institute of Molecular Medicine and Scleroderma Center, Thomas Jefferson University, Philadelphia, PA, United States of America.
PLoS One. 2018 May 2;13(5):e0196559. doi: 10.1371/journal.pone.0196559. eCollection 2018.
Transforming growth factor-β (TGF-β) plays a crucial role in the pathogenesis of Systemic Sclerosis (SSc) and other fibrotic disorders. TGF-β-mediated c-Abl and Src kinase activation induces strong profibrotic cascade signaling. The purpose of this study was to test in vivo the antifibrotic activity of Bosutinib (SKI-606), a second generation c-Abl and Src kinase inhibitor, on TGF-β induced cutaneous and pulmonary fibrosis. For this purpose, we employed the TBRIcaCol1a2Cre transgenic mice expressing an inducible constitutively active TGF-β receptor 1 constitutively activated by Col1a promoter-mediated Cre recombinase. The mice were treated parenterally with 2.5, 5.0 or 10.0 mg/kg/day of Bosutinib for 42 days. Skin and lungs from control and Bosutinib-treated mice (n = 6 per group) were assessed by histopathology, measurement of tissue hydroxyproline content, PCR analysis of tissue fibrosis associated gene expression, and evidence of myofibroblast activation. Mice with constitutive TGF-β-1 signaling displayed severe cutaneous and pulmonary fibrosis. Bosutinib administration decreased collagen deposition and hydroxyproline content in the dermis and lungs in a dose-dependent manner. Bosutinib also reversed the marked increase in profibrotic and myofibroblast activation-associated gene expression. These results demonstrate that constitutive TGF-β-1-signaling-induced cutaneous and pulmonary fibrosis were abrogated in a dose-related manner following parenteral administration of the c-Abl and Src tyrosine kinase inhibitor, Bosutinib. These results indicate that Bosutinib may be a potential therapeutic agent for tissue fibrosis in SSc and other fibroproliferative disorders.
转化生长因子-β(TGF-β)在系统性硬化症(SSc)和其他纤维化疾病的发病机制中起着关键作用。TGF-β 介导的 c-Abl 和Src 激酶激活诱导强烈的促纤维化级联信号。本研究旨在体内测试 Bosutinib(SKI-606),一种第二代 c-Abl 和Src 激酶抑制剂,对 TGF-β 诱导的皮肤和肺纤维化的抗纤维化活性。为此,我们使用了 TBRIcaCol1a2Cre 转基因小鼠,该小鼠表达由 Col1a 启动子介导的 Cre 重组酶组成型激活的可诱导组成型活性 TGF-β 受体 1。小鼠经皮下给予 2.5、5.0 或 10.0mg/kg/天的 Bosutinib 42 天。通过组织病理学、组织羟脯氨酸含量测量、组织纤维化相关基因表达的 PCR 分析以及肌成纤维细胞激活的证据,评估对照和 Bosutinib 治疗小鼠(每组 6 只)的皮肤和肺部。具有组成型 TGF-β-1 信号的小鼠表现出严重的皮肤和肺纤维化。Bosutinib 给药以剂量依赖性方式减少真皮和肺中的胶原蛋白沉积和羟脯氨酸含量。Bosutinib 还逆转了促纤维化和肌成纤维细胞激活相关基因表达的显著增加。这些结果表明,在皮下给予 c-Abl 和 Src 酪氨酸激酶抑制剂 Bosutinib 后,组成型 TGF-β-1 信号诱导的皮肤和肺纤维化以剂量相关的方式被消除。这些结果表明 Bosutinib 可能是 SSc 和其他纤维增生性疾病中组织纤维化的潜在治疗剂。