中国神经再生研究(英文版)

中国神经再生研究(英文版) ›› 2016, Vol. 11 ›› Issue (10): 1685-1694.doi: 10.4103/1673-5374.193251

• 原著:视神经损伤修复保护与再生 • 上一篇    下一篇

自体骨髓干细胞治疗Leber遗传性视神经病变：干细胞眼科治疗研究（SCOTS）

  

  • 收稿日期:2016-10-11 出版日期:2016-10-31 发布日期:2016-10-31

Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow-derived stem cells in the treatment of Leber’s hereditary optic neuropathy

Jeffrey N. Weiss1, Steven Levy2, *, Susan C. Benes   

  1. 1 Retina Associates of South Florida, 5800 Colonial Drive, Suite 300, Margate, FL, USA 2 MD Stem Cells, 3 Sylvan Road South, Westport, CT, USA 3 Te Eye Center of Columbus, Te Ohio State University, Columbus, OH, USA
  • Received:2016-10-11 Online:2016-10-31 Published:2016-10-31
  • Contact: Steven Levy, M.D., stevenlevy@mdstemcells.com.

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摘要：

干细胞眼科治疗研究（SCOTS）是目前为止ClinicalTrials.gov注册（Identifier NCT 01920867）的最大型临床试验眼科干细胞研究，SCOTS利用自体BMSC治疗视神经和视网膜疾病。根据患者的病情、视力丧失程度等给予患者包括眼球后、眼筋膜囊内、玻璃体内、视神经内、视网膜下注射及静脉灌注在内的干细胞移植，患者经治疗后ETDRS视力表测试的视力达到可看清35个符号，且Snellen视力从辨手动和辨指数改善至20/200和20/100，黄斑和视神经乳头神经纤维层明显增厚，视野缺损明显改善，治疗后无严重并发症发生。这些结果说明自体骨髓干细胞在治疗Leber遗传性视神经病变方面具有临床应用前景。 

orcid: 0000-0002-9313-3448 (Steven Levy)

关键词: 神经再生, Leber遗传性视神经病变, 线粒体疾病, 视神经病变, 骨髓干细胞, 失明, 视力丧失

Abstract: Te Stem Cell Ophthalmology Treatment Study (SCOTS) is currently the largest-scale stem cell ophthalmology trial registered at ClinicalTrials.gov (identifer: NCT01920867). SCOTS utilizes autologous bone marrow-derived stem cells (BMSCs) to treat optic nerve and retinal diseases. Treatment approaches include a combination of retrobulbar, subtenon, intravitreal, intra-optic nerve, subretinal, and intravenous injection of autologous BMSCs according to the nature of the disease, the degree of visual loss, and any risk factors related to the treatments. Patients with Leber’s hereditary optic neuropathy had visual acuity gains on the Early Treatment Diabetic Retinopathy Study (ETDRS) of up to 35 letters and Snellen acuity improvements from hand motion to 20/200 and from counting fngers to 20/100. Visual feld improvements were noted. Macular and optic nerve head nerve fber layer typically thickened. No serious complications were seen. Te increases in visual acuity obtained in our study were encouraging and suggest that the use of autologous BMSCs as provided in SCOTS for ophthalmologic mitochondrial diseases including Leber’s hereditary optic neuropathy may be a viable treatment option.

Key words: nerve regeneration, Leber’s hereditary optic neuropathy, mitochondrial disease, optic neuropathy, bone marrow derived stem cells, blindness, visual loss, neural regeneration