Departments of Physiology and Neuroscience, and Medicine, Zilkha Neurogenetic Institute, University of Southern California, Los Angeles, California, USA.
Travere Therapeutics, San Diego, California, USA.
JCI Insight. 2024 Sep 3;9(19):e177775. doi: 10.1172/jci.insight.177775.
Dual endothelin-1 (ET-1) and angiotensin II (AngII) receptor antagonism with sparsentan has strong antiproteinuric actions via multiple potential mechanisms that are more pronounced, or additive, compared with current standard of care using angiotensin receptor blockers (ARBs). Considering the many actions of ET-1 and AngII on multiple cell types, this study aimed to determine glomeruloprotective mechanisms of sparsentan compared to the ARB losartan by direct visualization of its effects in the intact kidney in focal segmental glomerulosclerosis (FSGS) using intravital multiphoton microscopy. In both healthy and FSGS models, sparsentan treatment increased afferent/efferent arteriole diameters; increased or preserved blood flow and single-nephron glomerular filtration rate; attenuated acute ET-1 and AngII-induced increases in podocyte calcium; reduced proteinuria; preserved podocyte number; increased both endothelial and renin lineage cells and clones in vasculature, glomeruli, and tubules; restored glomerular endothelial glycocalyx; and attenuated mitochondrial stress and immune cell homing. These effects were either not observed or of smaller magnitude with losartan. The pleiotropic nephroprotective effects of sparsentan included improved hemodynamics, podocyte and endothelial cell functions, and tissue repair. Compared with losartan, sparsentan was more effective in the sustained preservation of kidney structure and function, which underscores the importance of the ET-1 component in FSGS pathogenesis and therapy.
双重内皮素-1(ET-1)和血管紧张素 II(AngII)受体拮抗剂 sparsentan 通过多种潜在机制发挥强烈的抗蛋白尿作用,与目前使用血管紧张素受体阻滞剂(ARB)的标准治疗相比,这些机制更为明显或具有相加作用。鉴于 ET-1 和 AngII 对多种细胞类型的多种作用,本研究旨在通过使用活体多光子显微镜直接观察 sparsentan 在局灶节段性肾小球硬化(FSGS)中的完整肾脏中的作用,确定与 ARB 氯沙坦相比 sparsentan 的肾小球保护机制。在健康和 FSGS 模型中,sparsentan 治疗均增加了入球/出球小动脉直径;增加或保留了血流和单肾单位肾小球滤过率;减弱了急性 ET-1 和 AngII 诱导的足细胞钙增加;减少蛋白尿;保留足细胞数量;增加血管、肾小球和小管中的内皮和肾素谱系细胞和克隆;恢复肾小球内皮糖萼;并减弱线粒体应激和免疫细胞归巢。氯沙坦要么没有观察到这些作用,要么作用较小。sparsentan 的多效性肾保护作用包括改善血液动力学、足细胞和内皮细胞功能以及组织修复。与氯沙坦相比,sparsentan 在维持肾脏结构和功能方面更有效,这突显了 ET-1 成分在 FSGS 发病机制和治疗中的重要性。