参加首批干细胞视网膜疾病的临床试验显示改善视力

凤凰涅盘

在一个干细胞治疗视网膜疾病，两女的第一次临床试验，2012年1月24日 - - 干年龄相关性黄斑变性（AMD），其他与Stargardt病之一 - 表明，包括能够改善视力，治疗眼的视力表上读取多行。
最重要的是，治疗正在改进他们的独立性和执行日常活动的能力。干性AMD的78岁的女子说，她已经开始使她自己的早餐和购物。 Stargardt病人，在她50岁的平面设计师，报告周围的房子做更多的家务和再次骑着自行车。她还注意到，在她的色彩感知能力，以适应暗设置的改进。
每一种疾病之一 - - 朱尔斯斯坦在洛杉矶加州大学眼科研究所进行的两个I / II期临床试验，研究人员还与前两例患者的安全结果感到高兴。无不良事件时有发生，整体的安全性已被证明。四个月的研究结果报道，2012年1月23日，在杂志“柳叶刀”。
先进细胞技术（ACT），一家生物技术公司正在开发的治疗。该基金会战斗失明资助较早的干细胞实验室的研究，包括雷隆德博士的创新工作，这使得ACT的临床试验可能。
“这是一种很有前途的报告。斯蒂芬博士说​​：“我们非常高兴地得知治疗已安全迄今为止 - 在这些早期人类的研究，证明安全是最重要的的 - 我们很高兴，这两名患者都出现了改善视力，玫瑰，首席研究员主任，基金会的战斗失明。 “虽然仍然处于早期阶段的试验，结果是好消息，在美国超过10万的人，数以百万计的世界各地，视网膜变性疾病的影响。
“我们在该基金会已经兴奋的干细胞保存和恢复视力的潜力，以及ACT的结果表明，我们在正确的轨道。我们期待着更多的结果，从这个审判和未来基金会资助的临床试验，从干细胞的治疗。“
在这些早期阶段，ACT的治疗涉及50,000个视网膜色素上皮（RPE）细胞移植到每只眼睛移植。支持感光器，提供视觉细胞的视网膜色素上皮细胞，使用一个过程，不会破坏胚胎的人类胚胎干细胞衍生，发挥了关键作用。干性AMD和Stargardt病，视网膜色素上皮细胞退化，从而导致光感受器功能障碍和死亡。作为I / II期试验的进展，法案将在每个患者的视网膜移植的视网膜色素上皮细胞多达20万。 12人将参加这两项试验。
研究人员指出，在适当的位置视网膜的细胞移植到Stargardt病病人出现整合，并采取对正常的视网膜色素上皮的结构和组织。干性AMD患者的解剖结果不作为有利的视网膜色素上皮细胞没有整合。但研究人员鼓励他们提供改善视力。
博士罗斯指出，“我们必须牢记，这些结果与以往任何时候都从干细胞的一种疗法治疗视网膜疾病的前两个人。他们是非常短期的结果，所以有很多学习。随着中说，新闻是非常令人鼓舞，令人振奋。“
这些结果已在媒体广泛报道，包括本报告由英国广播公司新闻。

凤凰涅盘

1# 凤凰涅盘
Participants in the First Stem Cell Clinical Trials for Retinal Disease Show Improved Vision
anuary 24, 2012 – In the first-ever clinical trials of a stem cell treatment for retinal disease, two women – one with dry age-related macular degeneration (AMD), the other with Stargardt disease – demonstrated improved vision, including the ability to read more lines on an eye chart with their treated eyes.
Most important, the treatment is improving their independence and ability to perform daily activities. The 78-year-old woman with dry AMD says that she has begun making her own breakfast and shopping by herself. The Stargardt patient, a graphic designer in her fifties, reports doing more chores around the house and riding her bike again. She also notes improvements in her color perception and ability to adapt to dark settings.
Researchers from the Jules Stein Eye Institute at the University of California, Los Angeles, conducting the two Phase I/II clinical trials — one for each disease — are also pleased with the safety results for the first two patients. No adverse events have occurred, and overall safety has been demonstrated. Four-month results of the study were reported January 23, 2012, in the journal Lancet.
The treatment is being developed by Advanced Cell Technology (ACT), a biotechnology company. The Foundation Fighting Blindness funded earlier stem cell lab studies, including the innovative work of Dr. Ray Lund, which made ACT’s clinical trials possible.
“This is a promising report. We are very pleased to learn that the treatment has been safe thus far — demonstrating safety is paramount in these early human studies — and we are delighted that both patients are showing vision improvement,” says Dr. Stephen Rose, chief researcher officer, Foundation Fighting Blindness. “While the trials are still at an early stage, the results are good news for more than 10 million people in the U.S., and millions more around the world, affected by retinal degenerative diseases.
“We at the Foundation have been excited about the potential of stem cells for saving and restoring vision, and ACT’s results show that we are on the right track. We look forward to more results from this trial and future Foundation-funded clinical trials for treatments derived from stem cells.”
During these early stages, ACT’s treatment involves the transplantation of 50,000 retinal pigment epithelial (RPE) cells transplanted into each eye. The RPE cells, derived from human embryonic stem cells using a procedure that wouldn’t destroy the embryos, play a critical role in supporting photoreceptors, the cells that provide vision. In dry AMD and Stargardt disease, RPE cells degenerate, which leads to the dysfunction and death of photoreceptors. ACT will transplant as many as 200,000 RPE cells in each patient’s retina as the Phase I/II trials progress. Twelve people will be enrolled in each of the two trials.
Researchers note that the cells transplanted into the Stargardt disease patient appeared to integrate in the proper location of the retina and take on normal RPE structure and organization. The anatomical results for the dry AMD patient were not as favorable; the RPE cells did not integrate. But the researchers were encouraged by the vision improvement they appeared to provide.  
Dr. Rose notes, “We need to keep in mind that these results are for the first two people with a retinal disease ever to be treated with a therapy derived from stem cells. They are very short-term results, so there is a lot more to learn. With that said, the news is very encouraging and exciting.”
These results have been covered extensively in the media, including this report by BBC News.

雪山飞狐

谢谢；楼主的分享！

我一直在想一个问题，ACT公司治疗4个月就说安全，未免言之过早吧。

国内几个医院经过2年以上的观察都还没有说100%安全、

希望美国这个结果能够刺激中国的干细胞更快更安全的发展。

福娃晶晶

谢谢楼主！

凤凰涅盘

3# 雪山飞狐
我看了之后也比较疑惑，是不是翻译的问题？今天太忙，有时间再去ACT的网站上查一下。

muse

是不是跟北医三院马志中做的课题一样啊，视网膜色素上皮细胞移植治疗黄斑变性

南国乞儿

多谢楼主的分享.希望这样的消息来得更快些.

xiwang6666

希望国内的干细胞能够紧跟国际脚步,也希望今年的好消息不要像去年那样一出来h让大家兴奋一下又石沉大海了

chinatiger

5# 凤凰涅盘

这个实验的I期II期就是安全性实验，只是目前还不知道ACT以什么标准判断治疗是安全的。

凤凰涅盘

9# chinatiger
http://www.advancedcell.com/news ... institute/index.asp

下面是两篇来自ACT公司官网的文章是对上述内容的补充，各位战友可以研究一下。
ACT Announces that Additional Patient with Stargardt’s Disease Has Undergone Embryonic Stem Cell Transplantation at UCLA’s Jules Stein Eye Institute
MARLBOROUGH, Mass. — Jan. 25, 2012 – Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the the dosing of an additional patient in its Phase 1/2 trial for Stargardt’s macular dystrophy using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Tuesday (Jan. 24) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute. The patient successfully underwent the outpatient transplantation surgery and is recovering uneventfully.
“This is the fourth patient worldwide treated with ACT&rquo;s hESC-derived RPE cells,“ said Gary Rabin, chairman and chief executive officer of ACT. “We are pleased to be on schedule with our clinical programs to test the safety and tolerability of ACT&rquo;s stem cell-derived RPE cells. We are working with the best ophthalmology institutes to evaluate the capacity of our cell therapy which may have the ability to treat a variety of devastating diseases.”
The clinical trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. It is a prospective, open-label study, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt&rquo;s macular dystrophy at 12 months, the study&rquo;s primary endpoint. The patient, a 47-year-old male, was treated with the smallest dose of 50,000 cells. The hESC-derived RPE cells are currently also in Phase 1/2 for dry age-related macular degeneration. Preliminary results for the first two patients in each of the Phase 1/2 clinical trials were recently reported in The Lancet.
A third Phase1/2 trial for Stargardt’s macular dystrophy was recently initiated at the Moorfields Eye Hospital in London, treating the first patient on Jan. 20.
Stargardt&rquo;s Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. SMD causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium or RPE cell layer.
Further information about patient eligibility for the Stargardt&rquo;s macular dystrophy study is also available on www.clinicaltrials.gov
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit www.advancedcell.com.
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