系统性硬化症治疗的分子靶点。

Fibrogenesis & Tissue Repair Pub Date : 2012-06-06 eCollection Date: 2012-01-01 DOI:10.1186/1755-1536-5-S1-S19
Naoki Iwamoto, Oliver Distler
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引用次数: 17

摘要

尽管近年来在器官特异性治疗方面取得了重大进展,但迄今为止还没有批准的用于系统性硬化症(SSc)的“疾病改善”抗纤维化药物。尽管非选择性免疫抑制剂通常用于治疗SSc患者,但几乎所有免疫抑制剂都缺乏大型对照研究,需要进一步的证据来证明这些药物的长期有益作用。考虑到免疫抑制剂治疗SSc的这些事实,以及SSc的高死亡率,迫切需要其他治疗策略。最近报道了5-羟色胺(5-HT: 5-羟色胺)通路在纤维化中的重要作用。在这篇综述中,我们讨论5-HT在纤维化中的作用和该分子的治疗潜力。除了5-HT外,近年来还有许多有前景的靶点被广泛表征。对于许多这些分子靶点,临床研究中很容易获得修饰剂,而且这些修饰剂通常已经用于其他疾病的临床应用。这些研究的结果将表明,在多大程度上有希望的临床前结果,新的抗纤维化策略可以转化为临床实践。
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Molecular targets for therapy in systemic sclerosis.

Despite significant advances have been made in the recent years regarding organ-specific therapies, there is no approved 'disease-modifying' antifibrotic drug for systemic sclerosis (SSc) available to date. Although non-selective immunosuppressive agents are routinely used to treat patients with SSc, large well-controlled studies are lacking for almost all immunosuppressive agents and further evidence is required for long-term beneficial effects of these drugs. Considering these facts about immunosuppressive agents in SSc and also considering the high mortality of SSc, other therapeutic strategies are urgently needed. Recently an important role of the 5-hydroxytryptamine (5-HT: serotonin) pathway in fibrosis was reported. In this review, we discuss the role of 5-HT in fibrosis and therapeutic potential of this molecule. Besides 5-HT, there are a number of promising targets that have been extensively characterized in recent years. For many of these molecular targets, modifiers are readily available for clinical studies, and often these modifiers are used already in clinical use for other diseases. Results from these studies will show, in how far the promising preclinical results for novel antifibrotic strategies can be translated to clinical practice.

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