Batlle E, Pequerul R, Farrés J, Eriksson L A, Pors K, Jha V
Institute of Cancer Therapeutics, School of Pharmacy and Medical Sciences, Faculty of Life Sciences, University of Bradford, Bradford, BD71DP, UK.
Department of Biochemistry and Molecular Biology, Faculty of Biosciences, Universitat Autònoma de Barcelona, 08193, Bellaterra, Barcelona, Spain.
J Comput Aided Mol Des. 2025 Aug 31;39(1):72. doi: 10.1007/s10822-025-00622-3.
Aldehyde dehydrogenases (ALDHs) belong to a group of enzymes that play a vital role in various biological processes and cellular defence against aldehyde toxicity. The ALDH1A subfamily has largely been associated with elevated expression in cancer tissues, and in particular in cancer stem-like cells ALDH1A1 is a frequently expressed enzyme in stem cells and target for therapeutic intervention, however, other isoforms such as 1A2, 1A3, 2, 3A1 and 7A1 have drawn significant attention in the recent years due to their involvement in various pathophysiological conditions. The current study is aimed at therapeutic intervention of ALDH1A3 by developing new inhibitors with the aid of computer-assisted drug design approach. A mixed ligand- and structure-based virtual screening (VS) study employing 4 million compounds, was performed against the X-ray structure of ALDH1A3, supported by hierarchical and consensus docking employing 12 docking solutions, prediction of ADME properties, and binding free energy calculations, to identify new hit and selective compounds. One of the hit molecules (VS1) emerged as a new, and reversible inhibitor of ALDH1A3 from the biochemical screening and kinetic characterization. Molecular dynamics (MD) simulations further allowed us to distinguish between the protein-ligand dynamics of the VS hits and retinoic acid (RA) bound to ALDH1A3, establishing a correlation with the observed experimental results, further comparing with the reported ALDH1A3 inhibitors. VS1 can be used as a good starting point for structure-based hit optimization and hit-to-lead identification. The outcomes of this work are expected to benefit researchers working on computational and rational design of new ALDH inhibitors against cancer and other pathophysiological disorders.
醛脱氢酶(ALDHs)属于一组在各种生物过程以及细胞抵御醛毒性方面发挥至关重要作用的酶。ALDH1A亚家族在很大程度上与癌组织中表达升高有关,特别是在癌干细胞样细胞中。ALDH1A1是干细胞中频繁表达的一种酶,也是治疗干预的靶点。然而,近年来其他同工型,如1A2、1A3、2、3A1和7A1,因其参与各种病理生理状况而备受关注。当前的研究旨在借助计算机辅助药物设计方法开发新的抑制剂,对ALDH1A3进行治疗干预。针对ALDH1A3的X射线结构进行了一项混合的基于配体和结构的虚拟筛选(VS)研究,使用了400万种化合物,并辅以采用12种对接解决方案的分层和一致性对接、ADME性质预测以及结合自由能计算,以识别新的命中化合物和选择性化合物。从生化筛选和动力学表征中,一种命中分子(VS1)脱颖而出,成为ALDH1A3的一种新的可逆抑制剂。分子动力学(MD)模拟进一步使我们能够区分VS命中化合物与视黄酸(RA)与ALDH1A3结合时的蛋白质-配体动力学,与观察到的实验结果建立相关性,并进一步与报道的ALDH1A3抑制剂进行比较。VS1可作为基于结构的命中优化和从命中到先导物识别的良好起点。这项工作的成果有望使致力于针对癌症和其他病理生理疾病进行新的ALDH抑制剂的计算和合理设计的研究人员受益。