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jcyte最新消息

In the past few months, jCyte has made a number of important announcements. In December, we received an $8.3 million grant from the California Institute for Regenerative Medicine (CIRM). In April, we announced our phase 2b clinical trial. That same month, we received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food & Drug Administration (FDA). RMAT gives us better access to FDA expertise as we take our investigational retinitis pigmentosa (RP) therapy through clinical trials.

These announcements have generated interest from RP patients around the world and quite a few questions. We’d like to take this moment to give you the quick bullet on where jCyte is in the process and what all this means in the big picture.

The Therapy
Our investigational therapy (jCell) uses adult retinal progenitor cells to rescue rods and cones from RP progression and possibly generate new retinal cells. The treatment requires a single intravitreal injection, which can be performed in an ophthalmologist’s office with topical anesthetic. The entire procedure takes less than 30 minutes.

We have almost completed a phase 1/2a study to determine the therapy’s safety. Early results have been quite promising. So far, the treatment has been well-tolerated and has not generated a detectable immune response, a major concern when transplanting cells.

The Phase 2b Trial
On the strength of these results, jCyte has launched a phase 2b trial to test the treatment’s efficacy. The trial is enrolling 70 patients. After the trial is completed, we can determine next steps, which may include a phase 3 trial.

In addition to RMAT, jCell has received Orphan Drug designation from the FDA. These designations underscore the FDA’s interest in finding a therapy for RP, which currently has no medical treatments. The FDA and CIRM have been great partners in advancing jCell.

Many have asked how they can enroll in the clinical trial. Keep in mind, the study is being conducted exclusively in Southern California, and participants will need to come back to the clinic every three months to see if the therapy is working. If you have RP and are interested in the trial, you can send us an email or call 714-509-2151.

The Bottom Line
For people who have been waiting for an RP therapy for years or even decades, patience must seem like an obsolete concept. We get your emails and read your Facebook posts – you’re ready now.

However, we still need to go through the clinical trial process to determine whether jCell is an effective therapy. Our founders, Drs. Klassen and Yang, have been working on this problem for 20 years, and are determined to get jCell over the finish line.

How long will that take? We will know more soon. Stay tuned.
感觉可能是最近几年最有希望的疗法
感谢楼主带来的好消息!
在过去的几个月里,jCyte做了一些重要的公告。去年12月,我们收到了来自加州再生医学研究所(CIRM)的830万美元的拨款。今年4月,我们宣布了第2b阶段的临床试验。同月,我们接受了美国食品和药物管理局(FDA)的再生医学高级治疗(RMAT)。RMAT给我们提供了更好的获得FDA专业知识的途径,因为我们通过临床试验来接受我们的研究retinitis pigmentosa(RP)治疗。
  这些公告引起了世界各地RP患者的兴趣,也引起了不少问题。我们想借此机会告诉你jCyte在这一过程中所处的位置以及所有这些在大局中意味着什么。

  该疗法
  我们的研究治疗(jCell)使用***视网膜祖细胞从RP进展中挽救杆状和锥细胞,并可能产生新的视网膜细胞。这种治疗需要一种单一的静脉内注射,可以在眼科医生的办公室进行局部麻醉。整个过程只需不到30分钟。

  我们已经完成了一项1 / 2a的研究,以确定治疗的安全性。早期的结果是相当有希望的。到目前为止,治疗一直很耐受,并没有产生可检测的免疫应答,这是移植细胞的一个主要问题。

  2 b阶段试验

  在这些结果的强度上,jCyte已经启动了一个2b阶段的试验来测试治疗的功效。这项试验招收了70名患者。试验完成后,我们可以确定下一步,其中可能包括第三阶段试验。

  除了RMAT,jCell还获得了FDA的孤儿药。这些设计强调了FDA在寻找RP治疗方面的兴趣,目前没有医疗治疗。FDA和CIRM在推进jCell方面一直是很好的合作伙伴。

  许多人问他们如何才能参加临床试验。要记住,这项研究只在南加州进行,参与者需要每三个月回到诊所,看看治疗是否有效。如果你有RP并且对审判感兴趣,你可以给我们发邮件或打电话714 - 509 - 2151。

  底线

对于那些一直在等待RP治疗多年甚至几十年的人来说,耐心似乎是一个过时的概念。我们收到你的电子邮件并阅读你的Facebook帖子——你已经准备好了。

  然而,我们仍然需要通过临床试验来确定jCell是否是一种有效的治疗方法。我们的创始人,Drs。Klassen和Yang已经研究这个问题20年了,他们决心把jCell放在终点线上。

  需要多长时间?我们很快就会知道。请继续关注。
用翻译软件还是有很多问题的.比如干细胞是注射在玻璃体内的而不是静脉输入的
微信 xsfh550597973   QQ 550597973
谢谢楼主的好消息!看来jCyte正在有效的开展2期临床试验,正如文章结尾所说的那样“需要多长时间?我们很快就会知道”,希望就在眼前!
相信一切都会有可能。让我们持续地忍耐,不断地行进。
5# 雪山飞狐
呵呵,飞狐兄弟见笑了。俺没学过几天英语,又看不懂,只好用软件翻译过来看看是什么意思啦。总是希望出现真正的好消息哦。不好意思啦!以后希望战友们谁的英语基础好,给大家多解释解释国外的科研与临床治疗情况,那就方便了。
8# dfys 还是非常感谢你,
感谢楼主与Jcyte的联系并分享了他们的回复, 好战友!
2b期不知今年能否出结果,70个患者参与了试验,希望该医生所说的"很快"是今年,期待有惊喜的结果!
我是福建的RP战友,X性连锁遗传RPGR基因,自小夜盲且色弱,今年41岁,现在状况很糟,怕光且看人视物模糊有白雾,视野已管状,视力仅存50%,出门要人陪同,没工作收入,女儿5岁,高度散光且弱视,烦恼,急待治疗或助视器
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