Azienda Ospedaliero-Universitaria Ospedali Riuniti, Department of Medical and Neurological Sciences, Clinic of Nervous System Diseases, University of Foggia, Italy.
Neuropsychiatr Dis Treat. 2009;5:577-95. doi: 10.2147/ndt.s7788. Epub 2009 Nov 16.
Amyotrophic lateral sclerosis (ALS) is a relatively rare neurodegenerative disorder of both upper and lower motoneurons. Currently, the management of ALS is essentially symptoms-based, and riluzole, an antiglutamatergic agent, is the only drug for the treatment of ALS approved by the food and drug administration.
We reviewed current literature concerning emerging treatments for amyotrophic lateral sclerosis.
A Medline literature search was performed to identify all studies on ALS treatment published from January 1st, 1986 through August 31st, 2009. We selected papers concerning only disease-modifying therapy.
Forty-eight compounds were identified and reviewed in this study.
Riluzole is the only compound that demonstrated a beneficial effect on ALS patients, but with only modest increase in survival. Although several drugs showed effective results in the animal models for ALS, none of them significantly prolonged survival or improved quality of life of ALS patients. Several factors have been implicated in explaining the predominantly negative results of numerous randomized clinical trials in ALS, including methodological problems in the use of animal-drug screening, the lack of assessment of pharmacokinetic profile of the drugs, and methodological pitfalls of clinical trials in ALS patients.
肌萎缩性侧索硬化症（ALS）是一种相对罕见的上、下运动神经元的神经退行性疾病。目前，ALS 的治疗基本上是基于症状的，利鲁唑是唯一一种获得美国食品和药物管理局批准用于治疗 ALS 的抗谷氨酸药物。
我们回顾了有关肌萎缩性侧索硬化症新疗法的现有文献。
我们进行了 Medline 文献检索，以确定从 1986 年 1 月 1 日至 2009 年 8 月 31 日发表的关于 ALS 治疗的所有研究。我们只选择了关于疾病修饰疗法的论文。
本研究共确定并回顾了 48 种化合物。
利鲁唑是唯一一种对 ALS 患者有有益影响的化合物，但仅能适度延长生存时间。尽管有几种药物在 ALS 的动物模型中显示出有效结果，但没有一种能显著延长 ALS 患者的生存时间或改善生活质量。许多随机临床试验在 ALS 中主要得到阴性结果，有几个因素与之相关，包括在动物药物筛选中使用方法学问题、缺乏对药物药代动力学特征的评估，以及 ALS 患者临床试验中的方法学缺陷。
