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治疗肌萎缩侧索硬化症的潜在治疗药物和方法。

Potential therapeutic drugs and methods for the treatment of amyotrophic lateral sclerosis.

作者信息

Yacila G, Sari Y

机构信息

University of Toledo, College of Pharmacy and Pharmaceutical Sciences, Department of Pharmacology, Health Science Campus, 3000 Arlington Avenue, Toledo, OH 43614. USA.

出版信息

Curr Med Chem. 2014;21(31):3583-93. doi: 10.2174/0929867321666140601162710.

Abstract

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disorder caused by damage of motoneurons leading to paralysis state and long term disability. Riluzole is currently the only FDA-approved drug for the treatment of ALS. The proposed mechanisms of ALS include glutamate excitotoxicity, oxidative stress, mitochondrial dysfunction, protein aggregation, SOD1 accumulations, and neuronal death. In this review, we discuss potential biomarkers for the identification of patients with ALS. We further emphasize potential therapy involving the uses of neurotrophic factors such as IGFI, GDNF, VEGF, ADNF-9, colivelin and angiogenin in the treatment of ALS. Moreover, we described several existing drugs such as talampanel, ceftriaxone, pramipexole, dexpramipexole and arimoclomol potential compounds for the treatment of ALS. Interestingly, the uses of stem cell therapy and immunotherapy are promising for the treatment of ALS.

摘要

肌萎缩侧索硬化症(ALS)是一种神经退行性疾病,由运动神经元损伤引起,导致瘫痪状态和长期残疾。利鲁唑是目前唯一获得美国食品药品监督管理局(FDA)批准用于治疗ALS的药物。ALS的发病机制包括谷氨酸兴奋性毒性、氧化应激、线粒体功能障碍、蛋白质聚集、超氧化物歧化酶1(SOD1)蓄积和神经元死亡。在本综述中,我们讨论了用于识别ALS患者的潜在生物标志物。我们进一步强调了涉及使用神经营养因子(如胰岛素样生长因子I(IGFI)、胶质细胞源性神经营养因子(GDNF)、血管内皮生长因子(VEGF)、活性依赖的神经营养因子9(ADNF-9)、可力霉素和血管生成素)治疗ALS的潜在疗法。此外,我们描述了几种现有药物,如他拉喷酯、头孢曲松、普拉克索、右旋普拉克索和阿利克仑莫,它们是治疗ALS的潜在化合物。有趣的是,干细胞疗法和免疫疗法的应用前景广阔。

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