Wieder Nicolas, Greka Anna
Renal Division, Department of Medicine and Glom-NExT Center for Glomerular Kidney Disease and Novel Experimental Therapeutics, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA.
Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Pediatr Nephrol. 2016 Jul;31(7):1047-54. doi: 10.1007/s00467-015-3224-1. Epub 2015 Oct 21.
With more than 6,000 new pediatric patients with treatment-resistant nephrotic syndrome in the US each year alone, the unmet need for novel, podocyte-specific therapies is substantial. Recently, the established therapeutic benefit of angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARB) was used as a starting point to gain insight into the pathomechanism of primary podocytopathies. A calcium (Ca(2+))-mediated pathway has been identified that connects the angiotensin type 1 receptor (AT1R) to podocyte cytoskeletal dynamics, essential for a functioning glomerular filtration barrier. This discovery provided an important missing piece in our understanding of the pathomechanism of filter barrier damage, revealing Ca(2+) signaling as critical for podocyte health and disease. The identification of the two Ca(2+) permeant channels TRPC5 and TRPC6 as mediators of this pathway not only bolstered the importance of podocyte cytoskeleton dynamics but also revealed promising drug targets for treatment-resistant nephrotic syndrome. This review will focus on this novel signaling pathway in primary podocytopathies and its implications for next-generation therapies for glomerular disease.
仅在美国,每年就有超过6000名患有难治性肾病综合征的儿科新患者,对新型足细胞特异性疗法的需求尚未得到满足,这一需求十分巨大。最近,血管紧张素转换酶(ACE)抑制剂和血管紧张素受体阻滞剂(ARB)已确立的治疗益处被用作切入点,以深入了解原发性足细胞病的发病机制。已确定了一条钙(Ca(2+))介导的途径,该途径将1型血管紧张素受体(AT1R)与足细胞细胞骨架动力学联系起来,而这对于正常运作的肾小球滤过屏障至关重要。这一发现为我们理解滤过屏障损伤的发病机制提供了一个重要的缺失环节,揭示了Ca(2+)信号传导对足细胞健康和疾病至关重要。作为该途径介质的两个Ca(2+)渗透通道TRPC5和TRPC6的鉴定,不仅增强了足细胞细胞骨架动力学的重要性,还揭示了难治性肾病综合征有前景的药物靶点。本综述将聚焦于原发性足细胞病中的这条新信号通路及其对肾小球疾病下一代疗法的意义。