Lerner Christian, Jakob-Roetne Roland, Buettelmann Bernd, Ehler Andreas, Rudolph Markus, Rodríguez Sarmiento Rosa María
Pharmaceutical Research and Early Development (pRED), Roche Innovation Center Basel, F. Hoffmann-La Roche AG, Grenzacherstrasse 124, 4070 Basel, Switzerland.
J Med Chem. 2016 Nov 23;59(22):10163-10175. doi: 10.1021/acs.jmedchem.6b00927. Epub 2016 Oct 14.
A fragment screening approach designed to target specifically the S-adenosyl-l-methionine pocket of catechol O-methyl transferase allowed the identification of structurally related fragments of high ligand efficiency and with activity on the described orthogonal assays. By use of a reliable enzymatic assay together with X-ray crystallography as guidance, a series of fragment modifications revealed an SAR and, after several expansions, potent lead compounds could be obtained. For the first time nonphenolic and small low nanomolar potent, SAM competitive COMT inhibitors are reported. These compounds represent a novel series of potent COMT inhibitors that might be further optimized to new drugs useful for the treatment of Parkinson's disease, as adjuncts in levodopa based therapy, or for the treatment of schizophrenia.
一种专门针对儿茶酚-O-甲基转移酶的S-腺苷-L-甲硫氨酸口袋设计的片段筛选方法,使得能够鉴定出具有高配体效率且在所描述的正交测定中具有活性的结构相关片段。通过使用可靠的酶促测定并以X射线晶体学为指导,一系列片段修饰揭示了构效关系,经过几次扩展后,获得了有效的先导化合物。首次报道了非酚类且低纳摩尔强效的SAM竞争性COMT抑制剂。这些化合物代表了一系列新型强效COMT抑制剂,可能会进一步优化为可用于治疗帕金森病的新药,作为左旋多巴治疗的辅助药物,或用于治疗精神分裂症。