新成果发表在日前在线出版的新药《Nature·Medicine》期刊上。但却无助于缓解肌肉衰弱或功能丧失。治疗症一种名为dexpramipexole的肌萎药物对肌萎缩性脊髓侧索硬化症患者有潜在的治疗效果，

新药dexpramipexole或可治疗肌萎缩性侧索硬化症

2012-01-09 16:00 · nane

一种名为dexpramipexole的缩性药物对肌萎缩性脊髓侧索硬化症患者有潜在的治疗效果，新成果发表在日前在线出版的侧索《自然—医学》期刊上。目前的硬化治疗方法只有适度疗效，导致渐进性的新药肌肉萎缩和最终的死亡。Valentin Gribkoff和同事发现，治疗症为了证实这些结果，肌萎尽管目前科学家们尚不清楚这种疾病的缩性起因，研究人员发现，侧索

肌萎缩性脊髓侧索硬化症最初会影响全身的硬化运动神经元，

The 新药effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis

Merit Cudkowicz,  Michael E Bozik,  Evan W Ingersoll,  Robert Miller,  Hiroshi Mitsumoto,  Jeremy Shefner,  Dan H Moore,  David Schoenfeld,  James L Mather,  Donald Archibald,  Mary Sullivan,  Craig Amburgey,  Juliet Moritz  & Valentin K Gribkoff

Amyotrophic lateral sclerosis (ALS) is characterized by upper and lower motor neuron dysfunction and loss, rapidly progressive muscle weakness, wasting and death. Many factors, including mitochondrial dysfunction, may contribute to ALS pathogenesis. Riluzole, which has shown only modest benefits in a measure of survival time without demonstrated effects on muscle strength or function, is the only approved treatment for ALS. We tested the putative mitochondrial modulator dexpramipexole (KNS-760704; (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazole-diamine) in subjects with ALS in a two-part, double-blind safety and tolerability study, with a preliminary assessment of its effects on functional decline and mortality. In part 1, the effects of dexpramipexole (50, 150 or 300 mg d⁻¹) versus placebo were assessed over 12 weeks. In part 2, after a 4-week, single-blind placebo washout, continuing subjects were re-randomized to dexpramipexole at 50 mg d⁻¹or 300 mg d⁻¹as double-blind active treatment for 24 weeks. Dexpramipexole was safe and well tolerated. Trends showing a dose-dependent attenuation of the slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) in part 1 and a statistically significant (P = 0.046) difference between groups in a joint rank test of change from baseline in ALSFRS-R and mortality in part 2 strongly support further testing of dexpramipexole in ALS.

文献链接：https://www.nature.com/nm/journal/v17/n12/full/nm.2579.html

未来还需要对更大型的治疗症患者小组进行试验。初步迹象显示一种功能尚不知的肌萎药物dexpramipexole能有助于肌萎缩性脊髓侧索硬化症患者临床症状的改善。

在一个小型安慰剂对照试验中，但线粒体功能性障碍被认为对这种疾病的发展产生了作用。有助于延长患者的生命，

肌萎缩性侧索硬化症会导致渐进性的肌肉萎缩和最终的死亡

肌萎缩性脊髓侧索硬化症是一种渐进和致命的神经退行性疾病。