Drug Discovery, UCL Wolfson Institute for Biomedical Research, University College London, London WC1E 6BT, UK.
Department of Biological and Health Sciences, Universidad Loyola Andalucía, Dos Hermanas, Seville 41704, Spain.
Sci Adv. 2024 Jul 12;10(28):eado3501. doi: 10.1126/sciadv.ado3501. Epub 2024 Jul 10.
Macrocyclic drugs can address an increasing range of molecular targets but enabling central nervous system (CNS) access to these drugs has been viewed as an intractable problem. We designed and synthesized a series of quinolinium-modified cyclosporine derivatives targeted to the mitochondrial cyclophilin D protein. Modification of the cation to enable greater delocalization was confirmed by x-ray crystallography of the cations. Critically, greater delocalization improved brain concentrations. Assessment of the compounds in preclinical assays and for pharmacokinetics identified a molecule JP1-138 with at least 20 times the brain levels of a non-delocalized compound or those reported for cyclosporine. Levels were maintained over 24 hours together with low hERG potential. The paradigm outlined here could have widespread utility in the treatment of CNS diseases.
大环药物可以针对越来越多的分子靶点,但将这些药物递送到中枢神经系统 (CNS) 一直被视为一个棘手的问题。我们设计并合成了一系列针对线粒体亲环素 D 蛋白的新型 quinolium 修饰的环孢菌素衍生物。通过阳离子的 X 射线晶体学证实了阳离子的这种更大的离域化修饰。关键的是,更大的离域化改善了脑内浓度。通过临床前评估和药代动力学评估化合物,我们鉴定出一种名为 JP1-138 的化合物,其脑内水平至少是未离域化化合物或已报道的环孢菌素的 20 倍。该化合物在 24 小时内保持低 hERG 潜能的同时,维持着其脑内水平。这里概述的范例在治疗 CNS 疾病方面可能具有广泛的应用。