Aziz Ubair, Jabeen Ishrat
Computational Drug Design Lab, School of Interdisciplinary Engineering & Sciences (SINES), National University of Science & Technology (NUST), Islamabad, Pakistan.
PLoS One. 2025 Jun 4;20(6):e0324677. doi: 10.1371/journal.pone.0324677. eCollection 2025.
Transforming Growth Factor β (TGFβ) pathway has been recognized as one of the major processes involved in fibrotic diseases including the Atherosclerosis Cardiovascular Disease (ASCVD). Many drugs have been proposed and are under clinical trials for modulation of the TGFβ pathway by targeting TGFβ receptor. Recently, various proof of the concept studies have advocated that the inhibition of TGFβ-mediated Smad pathway could produce more focused effect with less off target toxicities in ASCVD. As these studies lack the mechanism and binding profile of Smad3 modulators, characterization of binding pattern for Smad3 inhibitors can provide a platform for the lead optimization against ASCVD. We utilized dimeric inhibitors from the PubChem dataset (PubChem ID: 630) of Smad3-FoxH1 binding inhibitors to generate binding hypothesis of Smad3 inhibitors. The selected compounds from the dataset were docked and ligand-protein complexes were simulated for 250 ns for further sampling of conformational space and to obtain stable binding hypothesis. Root Mean Square Deviation (RMSD), Root Mean Square Fluctuation (RMSF) and hydrogen bond analysis of ligand-protein complexes after simulations revealed that Asn320 in Smad3 provides an efficient inhibition site for the two most potent small inhibitors (hereby named SM1 and SM2) of Smad3-FoxH1 binding. Although the diverse nature of compounds produce variable interaction patterns with FoxH1 binding site in Smad3, Gln315, Gln364 and Arg367 were observed to be the most common hydrogen bond interaction points in this binding site. Additionally, two compounds (hereby named SM8 and SM19) detached from the FoxH1 binding site and formed a highly stable complex around Tyr323 via hydrophobic complementarity, suggesting a new binding site for modulation of Smad3 activity.
转化生长因子β(TGFβ)信号通路已被公认为是包括动脉粥样硬化性心血管疾病(ASCVD)在内的纤维化疾病所涉及的主要过程之一。目前已提出多种药物,并正在进行针对TGFβ受体调节TGFβ信号通路的临床试验。最近,各种概念验证研究主张,抑制TGFβ介导的Smad信号通路在ASCVD中可产生更具针对性的效果,且脱靶毒性更小。由于这些研究缺乏Smad3调节剂的作用机制和结合特征,表征Smad3抑制剂的结合模式可为针对ASCVD的先导化合物优化提供一个平台。我们利用来自Smad3 - FoxH1结合抑制剂的PubChem数据集(PubChem ID:630)中的二聚体抑制剂来生成Smad3抑制剂的结合假说。从数据集中选择的化合物进行对接,并对配体 - 蛋白质复合物进行250纳秒的模拟,以进一步采样构象空间并获得稳定的结合假说。模拟后对配体 - 蛋白质复合物的均方根偏差(RMSD)、均方根波动(RMSF)和氢键分析表明,Smad3中的Asn320为Smad3 - FoxH1结合的两种最有效的小分子抑制剂(在此命名为SM1和SM2)提供了一个有效的抑制位点。尽管化合物的多样性在Smad3中与FoxH1结合位点产生了可变的相互作用模式,但观察到Gln315、Gln364和Arg367是该结合位点最常见的氢键相互作用点。此外,两种化合物(在此命名为SM8和SM19)从FoxH1结合位点脱离,并通过疏水互补在Tyr323周围形成了高度稳定的复合物,这表明存在一个调节Smad3活性的新结合位点。