Laux Julian, Martorelli Mariella, Strass Simon, Schollmeyer Dieter, Maier Florian, Burnet Michael, Laufer Stefan A
Synovo GmbH, Paul-Ehrlich-Straße 15, Tübingen 72076, Germany.
Department of Pharmaceutical/Medicinal Chemistry, Eberhard Karls University Tübingen, Auf der Morgenstelle 8, Tübingen 72076, Germany.
ACS Pharmacol Transl Sci. 2023 Sep 21;6(10):1433-1452. doi: 10.1021/acsptsci.3c00119. eCollection 2023 Oct 13.
There is a general question in small molecule pharmacology about how apparent compound concentrations in blood, plasma, and organs actually relate to actual amounts at the target site of a compound. In this study, we used inherently fluorescent JAK3 ligands and their macrolide conjugates to investigate the relationship between physical properties, apparent bulk concentration, and organ and subcellular distribution. In vitro uptake into immune cells suggested that much of the substance was associated with granules or organelles. Samples from murine pharmacokinetic studies were analyzed by both conventional mass spectrometry and cryofluorescence microscopy methods to show the distribution of a compound within organs and cells without artifacts of fixation. These observations confirm the uptake of granules observed in vitro. Data from macrolides carrying either a coumarin fluorophore or a JAK3 inhibitor were similar, suggesting that the distribution is directed by the properties of the larger macrolide. These data show a propensity for azalide macrolides to concentrate in the lung and gut epithelia and suggest that the plasma- or whole-blood-derived estimates of drug levels almost certainly underestimate concentrations of macrolides in the mucous membranes. Thus, their apparent efficacy at sub-bacteriostatic doses may reflect their higher levels in barrier layers.
小分子药理学中存在一个普遍问题,即血液、血浆和器官中化合物的表观浓度实际上如何与化合物在靶点部位的实际含量相关。在本研究中,我们使用具有固有荧光的JAK3配体及其大环内酯缀合物来研究物理性质、表观总体浓度与器官及亚细胞分布之间的关系。免疫细胞的体外摄取表明,大部分物质与颗粒或细胞器相关。通过传统质谱法和低温荧光显微镜法对小鼠药代动力学研究的样本进行分析,以显示化合物在器官和细胞内的分布,而不存在固定假象。这些观察结果证实了体外观察到的颗粒摄取。携带香豆素荧光团或JAK3抑制剂的大环内酯的数据相似,表明分布是由较大大环内酯的性质决定的。这些数据显示氮杂内酯大环内酯有在肺和肠道上皮中浓缩的倾向,并表明血浆或全血衍生的药物水平估计几乎肯定低估了大环内酯在粘膜中的浓度。因此,它们在低于抑菌剂量时的表观疗效可能反映了它们在屏障层中的较高水平。