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FDA批准干性黄斑变性神经干细胞疗法的临床试验

  1. FDA
  2. 干性黄斑变性
  3. 神经干细胞疗法

来源:生物谷 2012-02-10 12:01

视网膜变性的一个突破性治疗将成为第二个进入Ⅰ/Ⅱ期临床试验的干细胞疗法。StemCells 公司(SCI)已获得美国食品和药物管理局授权对患干性-年龄相关性黄斑变性(AMD)病人开展神经干细胞治疗的临床试验。

视网膜变性的一个突破性治疗将成为第二个进入Ⅰ/Ⅱ期临床试验的干细胞疗法。StemCells 公司(SCI)已获得美国食品和药物管理局授权对患干性-年龄相关性黄斑变性(AMD)病人开展神经干细胞治疗的临床试验。

在先进细胞技术(ACT)之后10天,SCI发布这此消息,其中ACT发布了2011年视网膜变性的首个干细胞治疗临床试验,并宣布干性AMD和Stargardt病这两个首个研究参与者的鼓舞人心的安全与效能研究结果。SCI正计划开展干性AMD的临床试验,此临床试验由16家医院参与。此研究的其他细节即将出现。

SCI的神经干细胞疗法被设计成通过保护提供视力的视锥视杆细胞免于变性而维持视力。将在位于视网膜中心区域的黄斑下注射的治疗细胞被设计成持续释放保持视锥视杆细胞健康的蛋白。

在一篇发表于期刊 European Journal of Neuroscience的2012年1月30日版上的论文中, 研究人员报道了神经干细胞治疗方法在视网膜变性哺乳动物模型上的长期安全性与有效性。干细胞预防视力丧失,不引起任何免疫性反应或其他安全问题。

美国抗盲基金会首席研究专家Stephen Rose博士说,SCI治疗方法不同于ACT疗法的关键一点是:SCI的神经干细胞以它们天然的形式被移植以发挥保护作用。相反,ACT则从胚胎干细胞衍生视网膜色素上皮细胞(RPE)。新的RPE细胞被移植并替代、补充病人自身的RPE细胞,它是受如黄斑退行性变性一样的情况影响的。

"SCI的干细胞象一个药物工厂样发挥作用,这个工厂提供连续的蛋白递送以来保持视网膜健康并保持视力",他说,"另一方面,ACT则以替代疾病丧失的RPE为目标。RPE在视网膜中发挥关键性的支持与维持作用,为视锥视杆细胞提供营养与废物处理,ACT希望它的移植的RPE提供这些功能。希望两种方法都起作用。他们对治疗宽范围视网膜疾病有影响,包括视网膜色素变性和Usher综合症。"

俄勒冈健康与科学大学名誉教授、RSCI啮齿动物研究的研究人员Raymond Lund博士说,"如果没有美国抗盲基金会对我和我的团队的直接支持,将什么也不会发生,也就是说,美国抗盲基金会创造了一个环境,这个环境就是激励科学家们了解人们和他们的因视网膜疾病丧失视力的家人的需要。我很乐观,这项努力将给出视力获救的结果。"

近二十年来美国抗盲基金会资助各种项目,Lund博士在视网膜疾病的干细胞研究中是一名知名专家,在帮助ACT将其疗法推入临床试验中也起主导作用。

SCI在旧金山加利福尼亚大学进行一种胎儿神经疾病佩-梅病(Pelizaeus-Merzbacher disease)神经干细胞治疗方法的临床试验,并在瑞士开展脊髓损伤的临床研究。它也在从事阿尔茨海默症治疗方法的临床前研究。

美国抗盲基金会将公布SCI公司干性AMD临床试验包括招募信息、选入与淘汰标准在内的其他信息,一旦可用便会公布在网页上。(生物谷bioon.com)

Clinical Trial of Neural Stem Cell Treatment for Dry AMD Authorized by the FDA

Foundation Fighting Blindness/Feb. 7, 2012

A groundbreaking treatment for retinal degenerations is poised to become the second-ever stem cell therapy to move into a Phase I/II clinical trial. The company StemCells, Inc. (SCI), has received authorization from the FDA to launch a clinical trial of its neural stem cell treatment for people with the dry age-related macular degeneration (AMD).

SCI's announcement came 10 days after Advanced Cell Technology (ACT), which launched the first stem cell treatment clinical trials for retinal degenerations in 2011, announced encouraging safety and  efficacy  results for its first two study participants, one with dry AMD, the other with Stargardt disease. SCI is planning to launch a 16-participant clinical trial for dry AMD. Additional details of the study are forthcoming.

SCI's neural stem cell treatment is designed to preserve vision by protecting the rods and cones, the cells that provide vision, from degeneration. The therapeutic cells, which will be injected beneath the macula, the central region of the retina, are designed to continually release proteins that keep rods and cones healthy.

In a research paper published on January 30, 2012, in the European Journal of Neuroscience, researchers reported that the neural stem cell treatment was safe and effective over the long term in a rodent model of retinal degeneration. The stem cells prevented vision loss without causing any immunological reactions or other safety issues.

Dr. Stephen Rose, chief research officer at the Foundation Fighting Blindness, says that SCI's treatment differs from ACT's therapy in one key respect: SCI's neural stem cells are transplanted in their native form to play a protective role. In contrast, ACT derives retinal pigment epithelial (RPE) cells from embryonic stem cells. The new RPE cells are transplanted to replace and supplement the patients' own RPE cells, which are affected by conditions such as macular degeneration.

"SCI's stem cells works like a drug factory, providing continuous delivery of proteins to keep the retina healthy and preserve vision," he says. "ACT, on the other hand, has the goal of replacing RPE lost to disease. RPE play a critical support and maintenance role in the retina, providing nutrients and waste disposal for rods and cones, and ACT is hoping its transplanted RPE provide those functions as well. Hopefully, both approaches work. They have implications for treating a wide range of retinal conditions, including retinitis pigmentosa and Usher syndrome."

Dr. Raymond Lund, professor emeritus at the Oregon Health & Science University, and an investigator in the SCI rodent study, says, "None of this would have happened without direct Foundation support to me and my group, as well as the environment the Foundation has created to motivate scientists to be cognizant of the needs of people and their family members losing their sight to retinal diseases. I'm optimistic this effort will deliver sight-saving results."

Funded by the Foundation for various projects for nearly 20 years, Dr. Lund is a renowned expert in stem cell research for retinal diseases, and also played a leading role in helping ACT move its therapy into the clinic.

SCI has clinical trials underway at the University of California, San Francisco, for its neural stem cell treatment in Pelizaeus-Merzbacher disease, a fatal neural disorder in children, as well as a clinical study in Switzerland for spinal cord injury. It is also pursuing preclinical studies of its therapy in Alzheimer's disease.

The Foundation will post additional information about SCI's clinical trial for dry AMD, including recruiting contacts and inclusion and exclusion criteria, to its Web site when it becomes available.

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