Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, 1649-003 Lisbon, Portugal.
Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, 1649-003 Lisbon, Portugal.
Molecules. 2021 Sep 14;26(18):5574. doi: 10.3390/molecules26185574.
Multiple myeloma is an incurable plasma cell neoplastic disease representing about 10-15% of all haematological malignancies diagnosed in developed countries. Proteasome is a key player in multiple myeloma and proteasome inhibitors are the current first-line of treatment. However, these are associated with limited clinical efficacy due to acquired resistance. One of the solutions to overcome this problem is a polypharmacology approach, namely combination therapy and multitargeting drugs. Several polypharmacology avenues are currently being explored. The simultaneous inhibition of EZH2 and Proteasome 20S remains to be investigated, despite the encouraging evidence of therapeutic synergy between the two. Therefore, we sought to bridge this gap by proposing a holistic in silico strategy to find new dual-target inhibitors. First, we assessed the characteristics of both pockets and compared the chemical space of EZH2 and Proteasome 20S inhibitors, to establish the feasibility of dual targeting. This was followed by molecular docking calculations performed on EZH2 and Proteasome 20S inhibitors from ChEMBL 25, from which we derived a predictive model to propose new EZH2 inhibitors among Proteasome 20S compounds, and vice versa, which yielded two dual-inhibitor hits. Complementarily, we built a machine learning QSAR model for each target but realised their application to our data is very limited as each dataset occupies a different region of chemical space. We finally proceeded with molecular dynamics simulations of the two docking hits against the two targets. Overall, we concluded that one of the hit compounds is particularly promising as a dual-inhibitor candidate exhibiting extensive hydrogen bonding with both targets. Furthermore, this work serves as a framework for how to rationally approach a dual-targeting drug discovery project, from the selection of the targets to the prediction of new hit compounds.
多发性骨髓瘤是一种不可治愈的浆细胞肿瘤性疾病,占发达国家所有血液系统恶性肿瘤的 10-15%。蛋白酶体是多发性骨髓瘤的关键靶点,蛋白酶体抑制剂是目前的一线治疗药物。然而,由于获得性耐药,这些药物的临床疗效有限。克服这一问题的解决方案之一是多药理学方法,即联合治疗和多靶点药物。目前正在探索几种多药理学途径。尽管两种药物之间存在协同治疗的令人鼓舞的证据,但同时抑制 EZH2 和 20S 蛋白酶体仍然有待研究。因此,我们试图通过提出一种整体的计算策略来寻找新的双靶抑制剂来弥补这一空白。首先,我们评估了两个口袋的特征,并比较了 EZH2 和 20S 蛋白酶体抑制剂的化学空间,以确定双靶的可行性。随后,我们对 ChEMBL 25 中的 EZH2 和 20S 蛋白酶体抑制剂进行了分子对接计算,从中得出了一个预测模型,提出了在 20S 蛋白酶体化合物中寻找新的 EZH2 抑制剂的方法,反之亦然,这产生了两个双抑制剂命中。此外,我们为每个靶标构建了一个机器学习 QSAR 模型,但意识到它们在我们的数据中的应用非常有限,因为每个数据集占据了不同的化学空间区域。最后,我们对两种对接命中化合物针对两种靶标进行了分子动力学模拟。总的来说,我们得出结论,其中一种命中化合物作为双抑制剂候选物具有很大的潜力,它与两个靶标都有广泛的氢键。此外,这项工作为如何合理地进行双靶药物发现项目提供了一个框架,从靶标的选择到新的命中化合物的预测。