Competence Unit Molecular Diagnostics, Center Health and Bioresources, AIT - Austrian Institute of Technology GmbH, Vienna, Austria.
Handb Exp Pharmacol. 2021;265:75-110. doi: 10.1007/164_2020_370.
Knowledge about the transport of active compounds across the blood-brain barrier is of essential importance for drug development. Systemically applied drugs for the central nervous system (CNS) must be able to cross the blood-brain barrier in order to reach their target sites, whereas drugs that are supposed to act in the periphery should not permeate the blood-brain barrier so that they do not trigger any adverse central adverse effects. A number of approaches have been pursued, and manifold in silico, in vitro, and in vivo animal models were developed in order to be able to make a better prediction for humans about the possible penetration of active substances into the CNS. In this particular case, however, in vitro models play a special role, since the data basis for in silico models is usually in need of improvement, and the predictive power of in vivo animal models has to be checked for possible species differences. The blood-brain barrier is a dynamic, highly selective barrier formed by brain capillary endothelial cells. One of its main tasks is the maintenance of homeostasis in the CNS. The function of the barrier is regulated by cells of the microenvironment and the shear stress mediated by the blood flow, which makes the model development most complex. In general, one could follow the credo "as easy as possible, as complex as necessary" for the usage of in vitro BBB models for drug development. In addition to the description of the classical cell culture models (transwell, hollow fiber) and guidance how to apply them, the latest developments (spheroids, microfluidic models) will be introduced in this chapter, as it is attempted to get more in vivo-like and to be applicable for high-throughput usage with these models. Moreover, details about the development of models based on stem cells derived from different sources with a special focus on human induced pluripotent stem cells are presented.
关于活性化合物穿过血脑屏障的运输的知识对于药物开发至关重要。用于中枢神经系统(CNS)的系统应用药物必须能够穿过血脑屏障才能到达其靶位,而那些应该在周围发挥作用的药物则不应穿透血脑屏障,以免引发任何中枢不良反应。已经采用了多种方法,并开发了多种计算机模拟、体外和体内动物模型,以便能够更好地预测活性物质进入 CNS 的可能性。然而,在这种特殊情况下,体外模型起着特殊的作用,因为计算机模型的基础数据通常需要改进,并且必须检查体内动物模型的预测能力是否存在物种差异。血脑屏障是由脑毛细血管内皮细胞形成的动态、高度选择性的屏障。它的主要任务之一是维持 CNS 中的内稳态。屏障的功能由微环境中的细胞和血流介导的剪切应力调节,这使得模型开发变得最为复杂。一般来说,对于药物开发,人们可以遵循“尽可能简单,尽可能复杂”的信条来使用体外 BBB 模型。除了描述经典的细胞培养模型(transwell、hollow fiber)以及如何应用它们的指导之外,本章还将介绍最新的发展(球体、微流控模型),因为人们试图使这些模型更接近体内,并可适用于高通量使用。此外,还介绍了基于不同来源的干细胞开发模型的详细信息,特别关注人类诱导多能干细胞。