Neuralstem干细胞2期实验获FDA批准

菡萏尘雾 · 发表于 2013-4-25 14:47:03

本帖最后由菡萏尘雾于 2013-4-25 14:49 编辑

Maryland biotherapeutics company Neuralstem today announced that it has received approvalfrom the U.S. Food andDrug Administration (FDA) to conduct a phase 2 clinical trialto test its NSI-566 neural stem cells in people with amyotrophic lateral sclerosis (ALS).

马里兰生物医药公司Neuralstem今天（2013年4月17日）宣布他们获得美国食品和药物管理局（FDA）批准进行NSI-566神经干细胞针对ALS病人的2期临床实验。

Neural stem cells generate muscle-controlling nerve cells (motorneurons) and glia (a type of motor neuron support cell) inthe brain. It's hoped the experimental therapy will improve respiratoryfunction and prolong life in ALS.

神经干细胞生成控制肌肉的神经细胞（运动神经元）和神经胶质细胞（一种支持运动神经元功能的细胞）。希望这种实验性疗法可以提高ALS病人的呼吸功能，延长生命。

The trial, which is designed to assess safety and determine the maximum-tolerateddose, will expand to two centers:

该实验的设计目的是评估安全性，同时确定最大可耐受剂量。2期实验将拓展到两个中心进行：

· the MDA/ALS Center at Emory Universityin Atlanta(where an earlier phase 1 trial was conducted), with principal investigatorJonathan Glass; and

· 之前进行1期实验的亚特兰大埃默里大学MDA/ALS中心，首席研究者Jonathan Glass。

· the ALS Clinic at the University of Michigan Health System in Ann Arbor, with principal investigator Eva Feldman.

· 安阿伯市密歇根大学健康系统ALS门诊部，首席研究者Eva Feldman。

Determining the maximum-tolerated dose

确定最大可耐受剂量

Investigators expect to enroll 15 participants, who will be divided intofive different dosing groups. The first 12 participants will receive stem cellinjections into the cervical (neck) region of the spinal cord.The last three participants will receive injections in both the cervicaland lumbar (lower back)regions of the spinal cord.

研究人员预期招募15名参试者，分为5个不同的剂量组。最初的12名参试者将接受颈（脖）部脊髓干细胞植入。后面的3名参试者将进行颈部和腰椎（下后部）脊髓区域的干细胞植入。

In addition to other eligibility criteria, participants mustbe ambulatory, and must live within close geographic proximity to theresearch center at which they will participate.

除了其他合格标准外，参试者必须的可以走动（非卧床）的，住所要与研究中心比较接近。

"The aim of this phase 2 trial is to obtain the maximum-tolerateddose using the same route of administration as in phase 1, which was throughdirect injections into the gray matter of the spinal cord," NeuralstemChairman and Chief Scientific Officer Karl Johe saidin an April 17, 2013, Neuralstempress release."In phase 1, we started with just five injection sites per patient, andadvanced to a maximum of 15 injections of 100,000 cells each. In phase 2, wewill advance up to a maximum of 40 injections, and 400,000 cells per injectionbased on safety."

“2期实验的目的是获得最大的可耐受剂量，实验路径与1期一样，也就是直接向脊髓灰质内注射” Karl Johe（Neuralstem董事长兼首席科学家）2013年4月17日在新闻发布会上说到，“在1期实验中，我们从每位病人5个部位注射开始，随后增加到最多15个部位注射，每次100000细胞。2期实验中，我们将进一步增加到最多40次注射，以安全为前提，每次注射400000细胞”。

内容来源于：http://alsn.mda.org/news/fda-approves-als-stem-cell-trial

可参看：http://investor.neuralstem.com/p ... =1807567&highlight=