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  北京4月17日电(记者孙自法)国际知名学术期刊《自然》北京时间4月16日夜间同期在线发表两篇神经学论文称,研究人员通过开展两项独立的临床试验,展示出干细胞疗法对帕金森病的安全性。

  这两篇论文分别研究了利用人诱导多能干细胞和人类胚胎干细胞产生的细胞,对帕金森病的临床试验。后续,其研究还需要进一步来测试这些疗法的有效性和临床获益。

  据介绍,帕金森病是一种神经退行性疾病,其特征为逐渐丧失那些产生多巴胺(一种神经递质)的神经元。尽管目前的治疗方法如左旋多巴等能在早期减轻症状,但效果会逐渐下降,而且经常伴随着运动障碍(不自主运动)等副作用。细胞疗法,特别是补充脑部生产多巴胺的神经元(多巴胺能神经元)的疗法,或有望提供更有效、副作用更少的治疗方法。

  在其中一项研究中,为检验帕金森病细胞疗法的安全性和潜在副作用,论文共同通讯作者、日本京都大学高桥良辅(RyosukeTakahashi)和高桥淳(JunTakahashi)和同事进行了一项I/II期临床试验。7名患者(年龄在50-69岁)接受了多巴胺能前体细胞(来自人诱导多能干细胞)移植,区域为双侧大脑。在研究持续的24个月中,没有报告严重副作用,移植的细胞产生了多巴胺,没有过度生长或形成肿瘤(一种干细胞疗法的相关风险)。

  研究者还在继续参与试验进行有效性评估的6位患者中发现,有4位在未服用标准药物的情况下观察到帕金森病相关运动症状减少,有5位服药期间观察到相关运动症状减少,这些为研究的次要结果。但这些结果因测量而异,有些测量方法显示的变化极小。

  在另一项单独的I期临床试验中,论文第一作者兼通讯作者、美国纪念斯隆-凯特琳癌症中心V.Tabar和同事及合作者一起,研究了来自人胚胎干细胞的多巴胺能前体细胞产品的安全性。12名患者(年龄中位数为67岁)接受了手术移植多巴胺能前体细胞产品到双侧大脑壳核。5名患者接受了低剂量(每侧壳核90万细胞),7名接受了高剂量(每侧壳核270万细胞)。

  这些细胞产品的耐受普遍良好,在后续18个月中没有报告与疗法相关的严重副作用。没有出现运动障碍的迹象,该副作用此前与胚胎组织移植治疗帕金森病有关。此外,在低剂量和高剂量组均观察到一些运动功能的改善(研究的次要结果),但改善程度依不同测量参数而异。

  这两项临床试验都证实了异体(非自身)干细胞衍生细胞产品移植治疗帕金森病的安全性。不过,这两项研究存在一些局限性,包括样本量小,两项研究均为开放标签试验(研究者和患者都知道谁接受了何种类型的治疗)。

  《自然》同期还发表同行专家的“新闻与观点”文章认为,该两项独立试验都显示出安全性,并暗示可能有效,这朝着在更广泛社会中开展这种帕金森病细胞疗法迈出了重要一步。(完)新闻结尾 含羞待放 炼瓷:惊险瞬间曝光,背后故事让人意想不到!的相关文章
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