New drugs for ALS: How do we get there?

GlassJ.D.

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New drugs for ALS: How do we get there?

机译：ALS的新药：我们如何到达那里？

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Amyotrophic Lateral Sclerosis (ALS) is a disease that has perplexed investigators and patients since its initial description in the mid nineteenth century. The evidence regarding pathogenesis and progression of ALS does not point to a simple mechanism of disease. It is more than likely that the answer to the mystery of ALS pathogenesis will involve a combination of cellular abnormalities that are triggered by genetic predisposition and/or environmental insults. Ultimately, a balance of injury and the capacity for self-repair is tipped, leading to a physiological and structural disconnection of motor neurons from their targets, and manifesting as clinical weakness or spasticity. A rational approach for drug development for any disease requires some level of understanding of disease mechanisms. For ALS, the lack of a clear cause and the complexity of the experimental data on pathogenesis make this task quite challenging.

机译：自十九世纪中叶以来，肌萎缩性侧索硬化症（ALS）是一种引起研究者和患者困扰的疾病。关于ALS的发病机理和进展的证据并未指出疾病的简单机制。 ALS发病机理之谜的答案很有可能会涉及由遗传易感性和/或环境侮辱触发的细胞异常的组合。最终，损伤与自我修复能力之间的平衡得以实现，导致运动神经元与其靶标发生生理和结构上的分离，并表现为临床虚弱或痉挛。对任何疾病进行药物开发的合理方法都需要对疾病机理有一定程度的了解。对于ALS，缺乏明确的病因和有关致病机理的实验数据的复杂性使这项任务颇具挑战。

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《Experimental Neurology》 |2012年第1期|共6页
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GlassJ.D.;
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正文语种 eng
中图分类神经病学;
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New drugs for ALS: How do we get there?

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