Becker Daniel, Kaczmarska Zuzanna, Arkona Christoph, Schulz Robert, Tauber Carolin, Wolber Gerhard, Hilgenfeld Rolf, Coll Miquel, Rademann Jörg
Institute of Pharmacy, Medicinal Chemistry, Freie Universität Berlin, Königin-Luise-Straße 2+4, 14195 Berlin, Germany.
Institute for Research in Biomedicine, Parc Científic de Barcelona, Baldiri Reixac 10-12, 08028 Barcelona, Spain.
Nat Commun. 2016 Sep 28;7:12761. doi: 10.1038/ncomms12761.
Small-molecule fragments binding to biomacromolecules can be starting points for the development of drugs, but are often difficult to detect due to low affinities. Here we present a strategy that identifies protein-binding fragments through their potential to induce the target-guided formation of covalently bound, irreversible enzyme inhibitors. A protein-binding nucleophile reacts reversibly with a bis-electrophilic warhead, thereby positioning the second electrophile in close proximity of the active site of a viral protease, resulting in the covalent de-activation of the enzyme. The concept is implemented for Coxsackie virus B3 3C protease, a pharmacological target against enteroviral infections. Using an aldehyde-epoxide as bis-electrophile, active fragment combinations are validated through measuring the protein inactivation rate and by detecting covalent protein modification in mass spectrometry. The structure of one enzyme-inhibitor complex is determined by X-ray crystallography. The presented warhead activation assay provides potent non-peptidic, broad-spectrum inhibitors of enteroviral proteases.
与生物大分子结合的小分子片段可作为药物开发的起点,但由于亲和力低,往往难以检测。在此,我们提出了一种策略,通过其诱导靶标导向形成共价结合、不可逆酶抑制剂的潜力来鉴定与蛋白质结合的片段。一种与蛋白质结合的亲核试剂与双亲电弹头可逆反应,从而将第二个亲电试剂定位在病毒蛋白酶活性位点附近,导致酶的共价失活。该概念应用于柯萨奇病毒B3 3C蛋白酶,这是一种针对肠道病毒感染的药理学靶点。使用醛-环氧化物作为双亲电试剂,通过测量蛋白质失活率和在质谱中检测共价蛋白质修饰来验证活性片段组合。通过X射线晶体学确定了一种酶-抑制剂复合物的结构。所提出的弹头激活测定法提供了有效的非肽类肠道病毒蛋白酶广谱抑制剂。