内皮素系统作为心血管疾病的治疗靶点:厚望还是空想?
The endothelin system as a therapeutic target in cardiovascular disease: great expectations or bleak house?
作者信息
Kirkby N S, Hadoke P W F, Bagnall A J, Webb D J
机构信息
Centre for Cardiovascular Science, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, Scotland, UK.
出版信息
Br J Pharmacol. 2008 Mar;153(6):1105-19. doi: 10.1038/sj.bjp.0707516. Epub 2007 Oct 29.
There is considerable evidence that the potent vasoconstrictor endothelin-1 (ET-1) contributes to the pathogenesis of a variety of cardiovascular diseases. As such, pharmacological manipulation of the ET system might represent a promising therapeutic goal. Many clinical trials have assessed the potential of ET receptor antagonists in cardiovascular disease, the most positive of which have resulted in the licensing of the mixed ET receptor antagonist bosentan, and the selective ET(A) receptor antagonists, sitaxsentan and ambrisentan, for the treatment of pulmonary arterial hypertension (PAH). In contrast, despite encouraging data from in vitro and animal studies, outcomes in human heart failure have been disappointing, perhaps illustrating the risk of extrapolating preclinical work to man. Many further potential applications of these compounds, including resistant hypertension, chronic kidney disease, connective tissue disease and sub-arachnoid haemorrhage are currently being investigated in the clinic. Furthermore, experience from previous studies should enable improved trial design and scope remains for development of improved compounds and alternative therapeutic strategies. Although ET-converting enzyme inhibitors may represent one such alternative, there have been relatively few suitable compounds developed, and consequently, clinical experience with these agents remains extremely limited. Recent advances, together with an increased understanding of the biology of the ET system provided by improved experimental tools (including cell-specific transgenic deletion of ET receptors), should allow further targeting of clinical trials to diseases in which ET is involved and allow the therapeutic potential for targeting the ET system in cardiovascular disease to be fully realized.
有大量证据表明,强效血管收缩剂内皮素-1(ET-1)在多种心血管疾病的发病机制中起作用。因此,对ET系统进行药理学调控可能是一个有前景的治疗目标。许多临床试验评估了ET受体拮抗剂在心血管疾病中的潜力,其中最具阳性结果的试验使得混合ET受体拮抗剂波生坦以及选择性ET(A)受体拮抗剂西他生坦和安立生坦被批准用于治疗肺动脉高压(PAH)。相比之下,尽管体外和动物研究的数据令人鼓舞,但人类心力衰竭的治疗结果却令人失望,这或许说明了将临床前研究结果外推至人体的风险。目前,这些化合物在包括顽固性高血压、慢性肾病、结缔组织病和蛛网膜下腔出血等许多其他潜在应用方面正在进行临床研究。此外,以往研究的经验应有助于改进试验设计,并且仍有开发改进型化合物和替代治疗策略的空间。尽管ET转换酶抑制剂可能是一种替代方法,但开发出的合适化合物相对较少,因此,这些药物的临床经验仍然极其有限。随着近期的进展,以及通过改进的实验工具(包括ET受体的细胞特异性转基因缺失)对ET系统生物学的进一步了解,应该能够使临床试验进一步针对涉及ET的疾病,并充分实现ET系统在心血管疾病治疗中的潜力。