Long Keith, Kostman Stephen J, Fernandez Christian, Burnett James C, Huryn Donna M
Department of Pharmaceutical Sciences, University of Pittsburgh, Pittsburgh, Pennsylvania 15260, United States.
Ohio Wesleyan University, Delaware, Ohio 43015, United States.
ACS Med Chem Lett. 2019 Apr 24;10(6):1002-1006. doi: 10.1021/acsmedchemlett.9b00063. eCollection 2019 Jun 13.
The use of zebrafish in whole organism phenotypic assays has become a valuable strategy throughout the drug discovery process. Zebrafish assays can be used not only to screen libraries of compounds at the earliest stages but also to evaluate advanced leads for their effects on specific biological pathways or for toxicity. However, when confronted with inactivity of a compound in a zebrafish assay, there are little data that can be used to judge if the compound is truly biologically inert or inactive due to a lack of permeability into the model organism. While medicinal chemistry principles suggest parameters that are predictive of human oral bioavailability, cellular permeability, and even bacterial permeability, there have been no such parameters developed for zebrafish absorption. To address this question, we compiled a set of 700 compounds reported in the literature to be active in zebrafish assays, evaluated their properties, and compared them to properties derived from a set of historical drugs and a set of recently approved oral drugs. While some properties overlap, the averages and 10th and 90th percentiles of molecular weight, octanol-water partition coefficient (logP), H-bond counts, and polar surface area for zebrafish-active compounds are statistically different from those of known drugs. This analysis should be useful to scientists interpreting structure-activity relationships based on data from zebrafish assays and help to inform the translation from fish to mammals.
在整个药物发现过程中,斑马鱼用于全生物体表型分析已成为一种有价值的策略。斑马鱼分析不仅可用于在最早阶段筛选化合物库,还可用于评估先导化合物对特定生物途径的影响或其毒性。然而,当在斑马鱼分析中遇到化合物无活性时,由于缺乏进入模式生物的渗透性,几乎没有数据可用于判断该化合物是真正无生物活性还是由于缺乏渗透性而无活性。虽然药物化学原理提出了预测人体口服生物利用度、细胞渗透性甚至细菌渗透性的参数,但尚未开发出用于斑马鱼吸收的此类参数。为了解决这个问题,我们汇编了一组文献中报道的在斑马鱼分析中具有活性的700种化合物,评估了它们的性质,并将它们与一组历史药物和一组最近批准的口服药物的性质进行了比较。虽然有些性质重叠,但斑马鱼活性化合物的分子量、辛醇-水分配系数(logP)、氢键数和极性表面积的平均值以及第10和第90百分位数与已知药物在统计学上不同。该分析对基于斑马鱼分析数据解释构效关系的科学家应该是有用的,并有助于为从鱼类到哺乳动物的转化提供信息。