Tacconi Carlotta, Schwager Simon, Cousin Nikola, Bajic Davor, Sesartic Marko, Sundberg John P, Neri Dario, Detmar Michael
Institute of Pharmaceutical Sciences, Swiss Federal Institute of Technology, ETH Zurich, 8093 Zurich, Switzerland.
The Jackson Laboratory, Bar Harbor, Maine 04609, United States.
ACS Pharmacol Transl Sci. 2019 Aug 1;2(5):342-352. doi: 10.1021/acsptsci.9b00037. eCollection 2019 Oct 11.
Crohn's disease (CD) and ulcerative colitis (UC) are two distinct forms of inflammatory bowel disease (IBD) characterized by an expanded lymphatic network with impaired functionality both in mouse models and in human patients. In this study, we investigated whether targeted delivery of the pro-lymphangiogenic vascular endothelial growth factor C (VEGFC) to the site of inflammation may represent a new, clinically feasible strategy for treating IBD. To achieve targeting of inflamed tissue, we developed a fusion protein consisting of human VEGFC fused to the F8 antibody (F8-VEGFC), which specifically binds to the extradomain A (EDA) of fibronectin, a spliced isoform almost exclusively expressed in inflamed tissues. The therapeutic activity of intravenously administered F8-VEGFC, compared to a targeted construct lacking VEGFC (F8-SIP), was investigated in a mouse model of dextran sodium sulfate (DSS)-induced colitis. The presence of EDA fibronectin was detected in both human and mouse inflamed colon tissue. Biodistribution studies of radiolabeled F8-VEGFC revealed a specific accumulation of the antibody in the colon of DSS-administered mice, as compared to an untargeted VEGFC fusion protein (KSF-VEGFC) (binding the irrelevant hen egg lysozyme antigen). Systemic treatment with F8-VEGFC significantly reduced the clinical and histological signs of inflammation, expanded the lymphatic vascular network, reduced the density of immune cells, and also decreased the expression of inflammatory cytokines in the inflamed colon. Overall, these results reveal that administration of F8-VEGFC represents a novel and promising approach for the treatment of IBD.
克罗恩病(CD)和溃疡性结肠炎(UC)是炎症性肠病(IBD)的两种不同形式,其特征是在小鼠模型和人类患者中,淋巴管网络扩张且功能受损。在本研究中,我们调查了将促淋巴管生成的血管内皮生长因子C(VEGFC)靶向递送至炎症部位是否可能代表一种治疗IBD的新的临床可行策略。为了实现对炎症组织的靶向,我们开发了一种融合蛋白,该融合蛋白由与F8抗体(F8-VEGFC)融合的人VEGFC组成,F8抗体可特异性结合纤连蛋白的额外结构域A(EDA),EDA是一种几乎仅在炎症组织中表达的剪接异构体。在葡聚糖硫酸钠(DSS)诱导的结肠炎小鼠模型中,研究了静脉注射F8-VEGFC与缺乏VEGFC的靶向构建体(F8-SIP)相比的治疗活性。在人和小鼠的炎症结肠组织中均检测到EDA纤连蛋白的存在。放射性标记的F8-VEGFC的生物分布研究表明,与非靶向VEGFC融合蛋白(KSF-VEGFC)(结合无关的鸡蛋清溶菌酶抗原)相比,该抗体在给予DSS的小鼠结肠中特异性蓄积。用F8-VEGFC进行全身治疗可显著减轻炎症的临床和组织学体征,扩大淋巴管网络,降低免疫细胞密度,并降低炎症结肠中炎性细胞因子的表达。总体而言,这些结果表明,给予F8-VEGFC代表了一种治疗IBD的新颖且有前景的方法。