Fertala Jolanta, Romero Freddy, Summer Ross, Fertala Andrzej
1 Department of Orthopaedic Surgery, Sidney Kimmel Medical College, Thomas Jefferson University , Philadelphia, Pennsylvania.
2 Center for Translational Medicine and Jane and Leonard Korman Respiratory Institute, Thomas Jefferson University , Philadelphia, Pennsylvania.
Monoclon Antib Immunodiagn Immunother. 2017 Oct;36(5):199-207. doi: 10.1089/mab.2017.0044. Epub 2017 Oct 3.
Regardless of the cause of organ fibrosis, its main unwanted consequence is the formation of collagen fibril-rich deposits that hamper the structure and function of affected tissues. Although many strategies have been proposed for the treatment of fibrotic diseases, no therapy has been developed, which can effectively block the formation of collagen fibril deposits. With this in mind, we recently developed an antibody-based therapy to block key interactions that drive collagen molecules into fibrils. In this study, we analyzed target specificity, which is a main parameter that defines the safe use of all antibody-based therapies in humans. We hypothesized that, regardless of the route of administration, our antibody would preferentially bind to free collagen molecules synthesized at the sites of fibrosis and have minimal off-target interactions when applied in various tissues. To test this hypothesis, we used two experimental models of organ fibrosis: (1) a keloid model, in which antibody constructs were directly implanted under the skin of nude mice and (2) an experimental model of pulmonary fibrosis, in which our antibody was administered systemically by intravenous injection. Following administration, we studied the distribution of our antibody within target and off-target sites as well as analyzed its effects on fibrotic tissue formation. We found that local and systemic application of our antibody had high specificity for targeting collagen fibrillogenesis and also appeared safe and therapeutically effective. In summary, this study provides the basis for further testing our antifibrotic antibody in a broad range of disease conditions and suggests that this treatment approach will be effective if delivered by local or systemic administration.
无论器官纤维化的病因是什么,其主要的不良后果是形成富含胶原纤维的沉积物,这会妨碍受影响组织的结构和功能。尽管已经提出了许多治疗纤维化疾病的策略,但尚未开发出能够有效阻止胶原纤维沉积物形成的疗法。考虑到这一点,我们最近开发了一种基于抗体的疗法,以阻断驱动胶原分子形成纤维的关键相互作用。在这项研究中,我们分析了靶标特异性,这是定义所有基于抗体的疗法在人体中安全使用的一个主要参数。我们假设,无论给药途径如何,我们的抗体将优先结合在纤维化部位合成的游离胶原分子,并且在应用于各种组织时具有最小的脱靶相互作用。为了验证这一假设,我们使用了两种器官纤维化实验模型:(1)瘢痕疙瘩模型,其中将抗体构建体直接植入裸鼠皮下;(2)肺纤维化实验模型,其中通过静脉注射全身给药我们的抗体。给药后,我们研究了抗体在靶标和非靶标部位的分布,并分析了其对纤维化组织形成的影响。我们发现,局部和全身应用我们的抗体对靶向胶原纤维形成具有高度特异性,并且似乎安全且具有治疗效果。总之,这项研究为在广泛的疾病条件下进一步测试我们的抗纤维化抗体提供了基础,并表明这种治疗方法通过局部或全身给药将是有效的。