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NT-501营养因子最新进展

Neurotech's NT-501 Implant Demonstrates Statistically Significant Photoreceptor Preservation in Patients with Retinal Degenerative Disease
14 April 2011


Neurotech Pharmaceuticals today announced that, as reported in Investigative Ophthalmology & Visual Science (April, 2011, Vol. 52), the company's product NT-501 demonstrated statistically significant cone photoreceptor preservation in patients with retinitis pigmentosa

NT-501 is an intraocular implant that consists of human cells genetically modified to secrete ciliary neurotrophic factor (CNTF) - a growth factor capable of rescuing and protecting dying photoreceptors. The study utilized Adaptive Optics Scanning Laser Ophthalmoscopy (AOSLO), a breakthrough diagnostic technology, to image and measure the rate of the progressive degeneration of cone photoreceptors.

In the prospective study, two patients with RP and one patient with Usher syndrome type 2 were evaluated by AOSLO at 3, 6, 12, 18 and 24 months following implantation. For each patient, one eye received an NT-501 implant while the fellow eye received sham-treatment. The research team assessed photoreceptor loss by measuring cone density and comparing results for the active and sham treated eyes. No decrease in cone density was observed in any of the eyes treated with NT-501. A decrease in cone density are both indicative of photoreceptor loss.

In addition, the results demonstrated a statistically significant preservation of cone photoreceptors in the eyes of all three subjects treated with the NT-501 implant versus sham-treated eyes.

The study was led by Jacque Duncan, MD, Professor of Clinical Ophthalmology at the University of California. He said "We are extremely encouraged by the photoreceptor preserving effect of NT-501 seen in this study as well as the usefulness of AOSLO as a diagnostic tool for retinitis pigmentosa progression. Larger studies using AOSLO are urgently needed to confirm the photoreceptor protective effect f NT-501 treatment in patients with retinal degeneration."

This article is derived from Business Wire - an external news source - and RP Fighting Blindness accepts no responsibility for the content of linked external websites. The original source is linked below.
1

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下面文章大意 由风之子版主翻译

2011年4月14日 根据Neurotech公司的统计,NT501营养因子封装胶囊可以有效保护RP患者的视锥细胞。

在临床试验中,研究人员使用了自适应光学激光扫描眼底镜(AOSLO),以观察及测算患者的视锥细胞的退化速度。



在此次试验中,研究人员挑选了两个RP患者﹑一个2型尤塞尔(Usher)综合征患者,分别在植入NT501的3, 6, 12, 18 及 24个月后通过自适应光学激光扫描眼底镜对他们进行观察。试验采用双盲对比方法,通过对比植入NT501的眼睛及没有植入的眼睛内的视锥细胞的密度,研究人员发现,在上述患者的植入NT501的眼睛内没有发现视锥细胞密度下降。如果视锥细胞密度下降则表明感光细胞出现凋亡。



另外,试验的计算统计数据还表明,这三名患者植入NT501的眼睛的视锥细胞保存的很好。


试验负责人对结果非常满意,并且对使用自适应光学激光扫描眼底镜来观察RP患者的病情发展也非常满意。他们还将继续使用自适应光学激光扫描眼底镜来检测NT501的作用。
8# zhwei_313


有可能今年会和国内的医院合作多中心治疗,我正在咨询中。
9# 椰海


呵呵 这个胶囊是植入眼底的让它慢慢释放药物的,不是服用的胶囊。
国内的专家月底到美国开会,到时候会和美国机构谈合作事宜,大家耐心等待,期待好消息。
和国外机构合作也是参加多中心实验治疗,而不是正式开始治疗,实验治疗都是免费的。
我在网上看到的这篇文章,居然说有参加实验的RP患者视力提高了2到3行。

、神经保护治疗

1.        神经营养因子

视网膜细胞移植的成功使人想到可能是某些可扩散因子促进了光感受器细胞存活,这些因子即神经营养因子,它是一类能促进神经元存活、生长、分化.并维持其功能的细胞因子,它们的缺乏会加速光感受器细胞的变性死亡。用于RP研究的因子主要包括睫状神经营养因子(ciliary neurotrophic factor, CNTF)、脑源性神经营养因子(brain derived neurotrophic factor, BDNF)、成纤维细胞生长因子(basic fibroblast growth factor, bFGF)、胶质源性神经营养因子(glial-derived neurotrophic factor, GDNF)等,其疗效已在多种RP动物模型中得到验证[35-37]。但神经营养因子的半衰期较短,只能局部使用,多次玻璃体腔注射会引起许多并发症,因此治疗的关键在于选择有效的给药途径。CNTF基因修饰的人视网膜色素上皮细胞系(ARPE-19)通过细胞微囊技术植入RP患者眼内,6个月后取出微囊进行检测表明有大量活细胞分泌CNTF,参加实验的10个患者中有3眼术前视力低于20/800,研究期间视力未见下降;7眼视力提高,其中3眼提高10~15个字母,相当于Snellen视力表提高2~3行 [38]。这项研究结果表明,以病毒为载体的转基因治疗和细胞包囊技术联合基因工程技术是目前最
39# 福娃晶晶

美国的研究,就是这个主题帖的研究项目,我只知道是维持,不知道这个国内的眼科专家翻译以后说是有提高视力的。
41# 火光

现在的药物的效果都存在不确定性,你还是吃自己的药吧。
48# 凤凰涅盘

国内医院正在和这个公司商量参与实验治疗的事情,但是需要时间。
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