SCIENCE:干细胞疗法证实安全

2017-03-18 MedSci MedSci原创

人类首次来自诱导多能干细胞(IPS,Induced Pluripotent Stem Cells)分化的上皮细胞的临床试验终于出成果啦!

人类首次来自诱导多能干细胞(IPS,Induced Pluripotent Stem Cells)分化的上皮细胞的临床试验终于出成果啦!

2012年生物/医学诺贝尔奖得主山中伸弥(Shinya Yamanaka)通过导入外源基因的方法使体细胞去分化成为多能干细胞即诱导多能干细胞,高桥雅代(Masayo Takahashi)通过与其合作,找到了诱导的多能性干细胞生成视网膜上皮细胞的有效方法,并于2014年9月推进了人类首次来自诱导多能干细胞诱导的上皮细胞的临床试验,此项实验在一名七十多岁的老年性黄斑变性患者进行,该患者已经基本丧失了视力,而且也不太可能复原。这项倍受瞩目的实验经历1年的研究,终于公布初步成果。

此项研究评估了在患有新生血管性增龄性黄斑变性的患者体内移植一系列由诱导多能干细胞分化而来的视网膜色素上皮细胞的可行性。诱导多能干细胞来源于两名新生血管性增龄性黄斑变性晚期患者的表皮纤维母细胞,分化成视网膜色素上皮细胞,诱导多能干细胞和视网膜色素上皮细胞的来源性都经过广泛的检测。在其中一个患者体内,通过手术去除新生血管层并植入自体诱导多能干细胞分化的视网膜色素上皮细胞,术后1年,发现移植的一系列细胞还保存完整,最佳矫正视力既没有改善,也没有恶化,黄斑囊样水肿仍存在。

这篇由NEJM发表、由Science为其背书的研究成果说明:使用多能干细胞替代增龄性黄斑变性中被破坏的眼部组织,尽管并没有改善患者的视觉功能,但确实阻止了疾病的发展。作为使用多能干细胞进行人体内治疗手段的首创之举,其最大的意义在于:尽管有效性尚不确切,但证实了在人体内使用诱导多能干细胞是安全的,这是一个鼓舞人心的里程碑。此外,实验细节的发表有利于其他研究团队通过成熟组织源性的多能替代细胞来治疗病变或坏死组织。

在Science的文章中提到来自诱导多能干细胞分化的上皮细胞的临床试验中遇到的一些问题和进一步计划。第二名患者的实验过程中,多能干细胞出现不稳定情况,存在致癌的风险,基于安全考虑,实验中止。实验还在进一步推进,马上要进行的是已被批准的5个患者进行临床实验,一改之前进行每个患者自体多能干细胞诱导,高桥雅代她们将采取单一志愿者供体建立多能干细胞细胞库,并在技术成熟后应用于老年性黄斑变性早期患者。研究者和大众都对诱导多能干细胞充满希望,但现有实验都显示诱导多能干细胞有一定的帮助,但不能完全逆转疾病的发展,高桥雅代提醒大众对诱导多能干细胞的认识要现实,诱导多能干细胞可能并不如大家期望的那样有效。

原始出处:

Dennis Normile.  iPS cell therapy reported safe. Science   2017; 355, 1109-1110. DOI: 10.1126/science.355.6330.1109

Michiko Mandai,et al. Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration. N Engl J Med 2017; 376:1038-1046.DOI 10.1056/NEJMoa1608368.

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    2017-03-20 jichang
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    2017-03-19 1e10c84am36(暂无匿称)

    文章很好,值得分享

    0

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    2017-03-19 明月清辉

    谢谢分享,学习了

    0

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    2017-03-19 医艺依意

    每天快读顶级文献的感觉

    0

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    2017-03-19 刘煜

    学习新知识谢谢分享

    0

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