Lancet:胚胎干细胞首次被证实可用来治疗人类疾病
2014/10/16
继日本干细胞学者高桥雅代利用ips成功治疗眼睛疾病后,最近美国干细胞巨牛Robert Lanza证实利用胚胎干细胞可治疗老年黄斑变性和黄斑营养不良两种老年眼科疾病。该研究有望逐渐解冻相关部门对胚胎干细胞治疗的限制。

9月初,日本理化研究所(RIKEN)发育生物学中心的眼科专家高桥雅代使用诱导多能干细胞(iPS)治疗与年龄相关的视网膜退化疾病。近日,来自美国Advanced Cell Technology公司首席技术官Robert Lanza在Lancet杂志发表文章称,通过中长期的观察发现,胚胎干细胞治疗人体疾病是安全、有效的,这也是首个对胚胎干细胞治疗效果进行研究的报告。该研究有望加快干细胞在疾病治疗领域中的应用。

尽管胚胎干细胞研究是当前的热门领域,但是没有一份报告显示干细胞治疗人类疾病是安全和有效的。近日,Robert Lanza等人进行一项研究利用人体胚胎干细胞治疗两种老年性疾病,老年黄斑变性(age-related macular degeneration)和黄斑营养不良(macular dystrophy)。研究通过对18名患者直接移植胚胎干细胞中的视网膜上皮细胞后发现,患者的视力都先后得到了明显改善。胚胎干细胞1998年被发现,但是由于受到技术、伦理以及社会方面的限制或压力,对胚胎干细胞的研究一直鲜有进展,这项研究有望说服相关部门逐渐放开禁令,让胚胎干细胞造福更多患者。


胚胎干细胞移植逐渐改善眼疾

如同高桥雅代利用ips细胞治疗眼疾一样,Lanza也是从眼科疾病入手,这是因为干细胞具备在人体内转化成各种细胞的潜能,但它植入人体也有诱发畸形瘤和免疫抑制的可能,因此选择免疫豁免(immunoprivileged)位点,就显得非常重要,眼睛在这方面是一个不错的选择。

老年黄斑变性是老年人致盲的主要原因之一,主要发生在60岁以上的老人身上。黄斑营养不良(macular dystrophy)是一组有明确遗传因素的黄斑疾病,它是由于遗传性疾病所造成的早期或早熟细胞的病变与死亡,并无明确的病因可查。此种遗传影响可以在某种年龄出现,是由遗传决定的酶与代谢功能不全的结果。

奇人Lanza


国内可能对罗伯特•兰扎(Robert Lanza)很陌生,但是他早已经深入美国人内心,除了俊朗的外表令人印象深刻外,他还是一位极具个人魅力、颇具天分的科学家。2014年4 月 17 日,他在国际知名学术杂志 Cell Stem Cell 发表了一项突破性的干细胞研究,由他和韩国的一组研究人员利用体细胞核移植技术(Somatic cell nuclear transfer,简称 SCNT)成功地将成年男性表皮细胞克隆出胚胎干细胞,这是人类首次将成年人的体细胞克隆出胚胎干细胞。

在《时代周刊》2014 年评选的“100 位最具影响力人物”名单中,Lanza因首次利用成人体细胞克隆出胚胎干细胞入选。在干细胞研究领域,他的地位仅次于诺奖得主、日本学者山中伸弥。目前他已出版 30 多本书,发表上百篇学术论文。

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  • Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt’s macular dystrophy: follow-up of two open-label phase 1/2 studies

    Background Since they were first derived more than three decades ago, embryonic stem cells have been proposed as a source of replacement cells in regenerative medicine, but their plasticity and unlimited capacity for self-renewal raises concerns about their safety, including tumour formation ability, potential immune rejection, and the risk of differentiating into unwanted cell types. We report the medium-term to long-term safety of cells derived from human embryonic stem cells (hESC) transplanted into patients. Methods In the USA, two prospective phase 1/2 studies were done to assess the primary endpoints safety and tolerability of subretinal transplantation of hESC-derived retinal pigment epithelium in nine patients with Stargardt’s macular dystrophy (age >18 years) and nine with atrophic age-related macular degeneration (age >55 years). Three dose cohorts (50 000, 100000, and 150000 cells) were treated for each eye disorder. Transplanted patients were followed up for a median of 22 months by use of serial systemic, ophthalmic, and imaging examinations. The studies are registered with ClinicalTrials.gov, numbers NCT01345006 (Stargardt’s macular dystrophy) and NCT01344993 (age-related macular degeneration). Findings There was no evidence of adverse proliferation, rejection, or serious ocular or systemic safety issues related to the transplanted tissue. Adverse events were associated with vitreoretinal surgery and immunosuppression. 13 (72%) of 18 patients had patches of increasing subretinal pigmentation consistent with transplanted retinal pigment epithelium. Best-corrected visual acuity, monitored as part of the safety protocol, improved in ten eyes, improved or remained the same in seven eyes, and decreased by more than ten letters in one eye, whereas the untreated fellow eyes did not show similar improvements in visual acuity. Vision-related quality-of-life measures increased for general and peripheral vision, and near and distance activities, improving by 16–25 points 3–12 months after transplantation in patients with atrophic age-related macular degeneration and 8–20 points in patients with Stargardt’s macular dystrophy. Interpretation The results of this study provide the first evidence of the medium-term to long-term safety, graft survival, and possible biological activity of pluripotent stem cell progeny in individuals with any disease. Our results suggest that hESC-derived cells could provide a potentially safe new source of cells for the treatment of various unmet medical disorders requiring tissue repair or replacement.

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