Molecular targets for therapy in systemic sclerosis
Molecular targets for therapy in systemic sclerosis
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DOI：
10.1186/1755-1536-5-s1-s19
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发表时间：
2012-01-01
期刊：
影响因子：
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通讯作者：
Distler, Oliver
中科院分区：
文献类型：
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作者：
Iwamoto, Naoki;Distler, Oliver
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“疾病修饰”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，需要进一步证据证明这些药物的长期有益作用。考虑到免疫抑制剂在SSc中的这些事实以及SSc的高死亡率，迫切需要其他治疗策略。最近，5-羟色胺（5-HT：血清素）途径在纤维化中的重要作用被报道。在这篇综述中，我们讨论了5-HT在纤维化中的作用和这种分子的治疗潜力。除了5-HT，近年来还有许多有前途的靶点被广泛表征。对于许多这些分子靶点，修饰剂很容易用于临床研究，并且这些修饰剂通常已经用于其他疾病的临床使用。这些研究的结果将显示，在多大程度上有前途的临床前结果的新的抗纤维化策略可以转化为临床实践。
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.