新药dexpramipexole或可治疗肌萎缩性侧索硬化症
中国科学报 · 2012/01/09
一种名为dexpramipexole的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《自然—医学》期刊上。

肌萎缩性侧索硬化症

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。研究人员发现,一种名为dexpramipexole的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《Nature·Medicine》期刊上。

肌萎缩性脊髓侧索硬化症最初会影响全身的运动神经元,导致渐进性的肌肉萎缩和最终的死亡。尽管目前科学家们尚不清楚这种疾病的起因,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。目前的治疗方法只有适度疗效,有助于延长患者的生命,但却无助于缓解肌肉衰弱或功能丧失。

在一个小型安慰剂对照试验中,Valentin Gribkoff和同事发现,初步迹象显示一种功能尚不知的药物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。为了证实这些结果,未来还需要对更大型的患者小组进行试验。


参考文献

The 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−1 or 300 mg d−1 as 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:http://www.nature.com/nm/journal/v17/n12/full/nm.2579.html

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