Chaves Catarina, Do Tuan-Minh, Cegarra Céline, Roudières Valérie, Tolou Sandrine, Thill Gilbert, Rocher Corinne, Didier Michel, Lesuisse Dominique
Rare and Neurologic Diseases Research Therapeutic Area, Sanofi R&D, 91385 Chilly-Mazarin, France.
Translational Sciences Unit, Sanofi R&D, 91385 Chilly-Mazarin, France.
Pharmaceutics. 2020 Oct 14;12(10):967. doi: 10.3390/pharmaceutics12100967.
The non-human primate (NHP)-brain endothelium constitutes an essential alternative to human in the prediction of molecule trafficking across the blood-brain barrier (BBB). This study presents a comparison between the NHP transcriptome of freshly isolated brain microcapillaries and in vitro-selected brain endothelial cells (BECs), focusing on important BBB features, namely tight junctions, receptors mediating transcytosis (RMT), ABC and SLC transporters, given its relevance as an alternative model for the molecule trafficking prediction across the BBB and identification of new brain-specific transport mechanisms. In vitro BECs conserved most of the BBB key elements for barrier integrity and control of molecular trafficking. The function of RMT via the transferrin receptor (TFRC) was characterized in this NHP-BBB model, where both human transferrin and anti-hTFRC antibody showed increased apical-to-basolateral passage in comparison to control molecules. In parallel, eventual BBB-related regional differences were investigated in seven-day in vitro-selected BECs from five brain structures: brainstem, cerebellum, cortex, hippocampus, and striatum. Our analysis retrieved few differences in the brain endothelium across brain regions, suggesting a rather homogeneous BBB function across the brain parenchyma. The presently established NHP-derived BBB model closely mimics the physiological BBB, thus representing a ready-to-use tool for assessment of the penetration of biotherapeutics into the human CNS.
在预测分子跨血脑屏障(BBB)转运方面,非人灵长类动物(NHP)脑内皮是人类的重要替代模型。本研究比较了新鲜分离的脑微血管和体外筛选的脑内皮细胞(BECs)的NHP转录组,重点关注重要的BBB特征,即紧密连接、介导转胞吞作用的受体(RMT)、ABC和SLC转运蛋白,因为其作为预测分子跨BBB转运和识别新的脑特异性转运机制的替代模型具有重要意义。体外BECs保留了大部分用于维持屏障完整性和控制分子转运的BBB关键元件。在这个NHP - BBB模型中,通过转铁蛋白受体(TFRC)对RMT的功能进行了表征,与对照分子相比,人转铁蛋白和抗hTFRC抗体在该模型中均显示出从顶侧到基底侧的转运增加。同时,对来自脑干、小脑、皮质、海马体和纹状体这五个脑结构的体外筛选7天的BECs中可能存在的与BBB相关的区域差异进行了研究。我们的分析发现不同脑区的脑内皮之间差异很少,这表明整个脑实质的BBB功能相当均匀。目前建立的源自NHP的BBB模型紧密模拟了生理性BBB,因此是评估生物治疗药物进入人类中枢神经系统渗透性的现成工具。