Bassi Roberto, Fornoni Alessia, Doria Alessandro, Fiorina Paolo
Division of Nephrology, Boston Children's Hospital, Harvard Medical School, 300 Longwood Ave., Enders Building, Boston, MA, 02115, USA.
Department of Transplant Medicine, IRCCS Ospedale San Raffaele, Milan, Italy.
Diabetologia. 2016 Jan;59(1):21-29. doi: 10.1007/s00125-015-3766-6. Epub 2015 Sep 26.
Diabetic kidney disease (DKD) is the leading cause of end-stage renal disease in the Western world. Standard treatments have ultimately proven ineffective in blocking DKD progression, thus necessitating the design of new therapies to complement glycaemic and blood pressure control. High glucose levels upregulate the immune-related molecule B7-1 in podocytes, and such an event may play a relevant role in DKD onset, suggesting that B7-1 is a suitable therapeutic target for DKD. CTLA4-Ig is a clinically available fusion protein, approved for the treatment of some autoimmune diseases, which binds B7-1 and blocks its signalling. We have previously demonstrated that CTLA4-Ig restores the physiological structure and cellular motility of podocytes challenged with high glucose in vitro and abrogates the onset of proteinuria in murine models of DKD in vivo. Notably, these beneficial effects occurred independently of any systemic immunological effects of CTLA4-Ig. While the expression of B7-1 on podocytes raises questions regarding the very nature of the podocyte as we know it, the preliminary positive effect of CTLA4-Ig on proteinuria in preclinical models and the evidence of B7-1 expression in kidney biopsies of diabetic individuals suggest a potential novel indication for CTLA4-Ig in DKD. Nonetheless, recent reports of problems with detecting podocyte B7-1 and of inconsistent therapeutic efficacy of CTLA4-Ig in proteinuric patients highlight the necessity to establish uniformly accepted protocols for the detection of B7-1 and underline the need for randomised trials with CTLA4-Ig in kidney diseases.
糖尿病肾病(DKD)是西方世界终末期肾病的主要病因。标准治疗最终被证明无法有效阻止DKD的进展,因此需要设计新的疗法来辅助血糖和血压控制。高血糖水平会上调足细胞中与免疫相关的分子B7-1,这一事件可能在DKD的发病中起重要作用,表明B7-1是DKD合适的治疗靶点。CTLA4-Ig是一种临床可用的融合蛋白,已被批准用于治疗某些自身免疫性疾病,它能结合B7-1并阻断其信号传导。我们之前已经证明,CTLA4-Ig可恢复体外高糖刺激下足细胞的生理结构和细胞运动能力,并在体内DKD小鼠模型中消除蛋白尿的发生。值得注意的是,这些有益效果的出现与CTLA4-Ig的任何全身免疫效应无关。虽然足细胞上B7-1的表达引发了我们对足细胞本质的质疑,但CTLA4-Ig在临床前模型中对蛋白尿的初步积极作用以及糖尿病个体肾活检中B7-1表达的证据表明,CTLA4-Ig在DKD中可能有新的应用指征。尽管如此,最近关于检测足细胞B7-1存在问题以及CTLA4-Ig在蛋白尿患者中治疗效果不一致的报道强调了建立统一认可的B7-1检测方案的必要性,并突出了在肾脏疾病中对CTLA4-Ig进行随机试验的必要性。