法国 RdCVF 疗法将进入临床实验

雪山飞狐

法国凹制药公司的RdCVF疗法已经获得了欧洲药物监管机构（类似美国FDA的）的批准进入临床实验阶段，并且获得了孤儿药资格，以便可以更快的进行实验和进入临床应用。

RdCVF疗法主要是保护视网膜视锥细胞，视锥信主要负责中央视力和白天视野。

明年的第一期临床实验将是以眼底注射的形式治疗，每月注射一次，以后研究者将研究用基因疗法使药物能够长期的在视网膜发挥作用。


French Researchers Planning Clinical Study of Vision-Preserving Protein
A promising treatment aimed at preserving cones, the retinal cells that provide central and daytime vision, is poised to move into a Phase I clinical trial within the next year. Known as rod-derived cone viability factor (RdCVF), the therapeutic protein has consistently preserved vision in several preclinical studies.

RdCVF has received an orphan medicinal product designation from the European Commission, a regulatory agency similar to the FDA in the U.S. The orphan designation provides marketing, financial, and clinical research benefits to Fovea Pharmaceuticals, the French company developing the treatment.

In 2005, Drs. José-Alain Sahel and Thierry Léveillard received the Foundation’s Annual Trustee Award for their discovery of RdCVF as a potential vision-saving treatment. The Foundation-funded French research team screened 210,000 genes to find a rod-derived protein that would protect cones.

A majority of retinal degenerative diseases, including retinitis pigmentosa (RP), are caused by mutations in genes that affect rod cells. As a result, rods are the first photoreceptors to degenerate. Rods provide peripheral eyesight and vision in dark settings.

However, once rods are gone, cones subsequently degenerate. This phenomenon led researchers to suspect that rods were secreting a factor (or multiple factors) that helped to preserve cones.

Initially, the France-based clinical trial will involve monthly ocular injections of RdCVF into people with RP. The investigators are also evaluating gene therapy as a delivery mechanism to provide long-term, sustained release of RdCVF. One gene therapy treatment will likely be effective for several years.

While the researchers will initially evaluate RdCVF in people with RP, they believe the treatment may preserve vision in people with a wide range of retinal degenerative diseases.

Dr. Sahel notes that by keeping as few as 5 percent of cones alive, a person can continue to function independently.

Drs. Sahel and Léveillard are co-founders of Fovea Pharmaceuticals. Dr. Sahel is center director for the Foundation’s Paris Research Center for the Study of Retinal Degenerative Diseases.

雪山飞狐

可能大家对这个消息不是太敢兴趣，认为这个治疗离我们太远，没有实际意义。

可是现在一些国际上的研究项目都是国际性多中心的实验，国内的医院都可以参加的，最多是等到2期实验的时候就可以合作了。

我知道有一家国内权威的医院已经和国外的研究机构合作开展了一个药物方面的合作，明年还会有一个合作项目。

因此我对国内的医院也慢慢感兴趣了。

雪山飞狐

4# 罗兰德尼奥

这个不是那个胶囊技术，完全不同的技术。

那个生长因子胶囊技术治疗RP，明年将会进入第三期，有可能会在中国的一家医院进行。

雪山飞狐

8# acon168

是针对RP的，针对RP中的视锥细胞的。

雪山飞狐

14# yuxin

这个药物就是针对视锥细胞而设计的，是一种视锥细胞的活性因子，那对视锥细胞营养不良肯定有帮助的。

雪山飞狐

呵呵  看把罗兰急得，这个实验是第一期，但是你要知道这个治疗方法已经获得了孤儿药的资格，可以加快实验和上市的速度，而且到了二期和三期国内的医院就有可能参加他们的合作了。

雪山飞狐

下面一段是这个疗法通过基因给药的方式治疗，这样的方法使药物持续的时间更长和效果更好。虽然只是动物实验结果，但是科学家认为这种治疗方法对恢复视锥细胞功能有很大的帮助，这篇文章发表在2010.6.24的Science杂志上。


An international research team, funded in part by the Foundation Fighting Blindness, has used gene therapy to reactivate retinal cells that were previously unresponsive to light. The treatment was delivered to cones, the retinal cells that provide central and daytime vision. Investigators evaluated the gene therapy in two mouse models of retinal degenerative disease, as well as cultures of human retinal tissue. Using a variety of tests, the researchers demonstrated that the treated cells restored functional vision, and that the restoration of vision persisted over a long period of time.

In retinal degenerative diseases such as retinitis pigmentosa, cones stop providing vision before they die and can remain in a compromised state for several years. The gene therapy used to restore vision in the cone-reactivation study leads to the production of a light-sensitive protein called halorhodopsin, which is similar to vision-activating proteins occurring naturally in the retina. The presence of halorhodopsin appears to reactivate the visual cycle, the biochemical process in retinal cells that lead to vision.

An adeno-associated virus (AAV), a therapeutic virus, was used to deliver the treatment to the cones. AAVs are the same delivery mechanism used in landmark gene therapy clinical trials that have restored vision in 28 children and young adults who were virtually blind from Leber congenital amaurosis.

Dr. José-Alain Sahel, one of the cone-reactivation study investigators and director of the Foundation-funded Paris Research Center for the Study of Retina Degenerative Diseases, says that through high-resolution imaging, his team has identified people who may be good candidates for a future clinical trial. He adds that even people who are legally blind might have some cones reactivated and vision restored.

Dr. Sahel also notes that the cone-reactivation treatment has the potential to be even more beneficial if coupled with a therapy that helps protect cones from degeneration. Dr. Sahel and his team are planning a clinical trial of a neuroprotective treatment called rod-derived cone viability factor, or RdCVF, which has shown excellent results for preserving cones in preclinical studies.

The cone-reactivation study was led by Dr. Botond Roska of the Friedrich Miescher Institute for Biomedical Research in Switzerland, and included scientists from several European research institutions. Results of the study were published in the June 24 issue of the journal Science

雪山飞狐

补充：这个RdCVF 疗法是美国抗盲基金会赞助的，抗盲基金会有很充足的资金赞助世界上各种治疗RP的多中心实验，他们还赞助了我们中国的基因检测项目和一些临床实验项目（运作中）。