Thirteen Participants Treated in ACT’s Stem Cell Clinical Trial
Nov. 15, 2012 – Results from the first-ever clinical trials of a retinal disease treatment derived from stem cells continue to be encouraging. Advanced Cell Technology (ACT), developer of the therapy, recently reported that 13 people — nine with Stargardt disease and four with dry age-related macular degeneration (AMD) — have had no safety issues after receiving transplanted cells. In addition, modest vision improvements for the first participants treated more than a year ago have been sustained.
According to ACT, the transplanted cells — retinal pigment epithelial (RPE) cells developed from human embryonic stem cells — appear to be engrafting into the retinas of some of the participants. Engraftment is an indicator that the transplanted cells have the potential to function like native cells.
RPE cells provide essential nutrition and waste removal for photoreceptors, the cells that provide vision. The dysfunction of RPE cells in dry AMD and Stargardt disease eventually leads to loss of photoreceptors.
The first nine participants in ACT’s trials each received transplants of 50,000 cells, and the next four received 100,000 cells. The researchers plan to transplant as many as 200,000 cells for the treated retina of each patient during their three Phase I/II clinical trials. Two studies — one for dry AMD, the other for Stargardt disease — are taking place in the United States. A trial for Stargardt disease is also underway in the United Kingdom.
ACT’s researchers plan to treat people with less advanced disease in later stages of their human studies. They believe the treatment may provide greater vision improvement in these individuals.
The Foundation Fighting Blindness funded laboratory research that made ACT’s human studies possible.
摘要：
* Investigators for the Company’s Phase I/II clinical trials for Stargardt’s macular dystrophy (SMD) and dry age-related macular degeneration (dry AMD) have observed evidence of engraftment of the transplanted human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells and visual acuity gain in patients treated over the 18 months since the trials were first initiated.
经查，在针对干性黄斑萎缩和斯特格病变的临床试验I/II期中，有证据表明移植的人体胚胎干细胞已定植，且自植入之后18个月内，促生试验病患的RPE细胞并促其获得视力（此处没有说明，但我记得柳叶刀里面提到应该是部分视力，表现为读取字数的增多）。
* The Company believes this observation of engraftment and persistence is clinically important because engraftment of cells in the proper area, and recapitulation of normal RPE cell layer structure is important to the ultimate efficacy of the RPE cells in the treatments.
公司认为这定植和留存的观察结果具有临床重大意义，因为这表明植入的区域是正确的，并且在治疗中重演正常RPE细胞表层结构对最终RPE细胞的有效性是相当重要的。
* Additionally, investigators have not observed any issues of safety relating to the transplanted human embryonic stem cell (hESC)-derived RPE cells in any of the 18 patients treated. The company believes this warrants early (though cautious) optimism as the company looks forward to initiating the second half of the three trials.
此外，在对已经进行的18例病患植入中，调查没有发现相关的安全性问题。尽管仍然谨慎，但公司相信在即将进行另半临床试验中，这是一个乐观的信号。
