FDA批准UCLA的IND申请，进行胚胎干细胞的严重近视眼临床试验

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美国FDA日前批准了由UCLA和ACT合作进行的人胚胎干细胞治疗严重近视的新药临床申请。此项研究将采用人胚胎干细胞来源的视网膜色素上皮细胞RPE进行12例为期1年的临床试验，目的是检验治疗的安全性和耐受性。. @0 I5 I  t; O2 W; E( A5 ~
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FDA approves UCLA IND application to commence embryonic stem cell-based trial
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" G# q" ?- p- U0 s" ]" DPublished 12 February 2013, @9 }. _4 {  g5 S% v6 f( C' S

. V' h! b! u  @4 gThe USFDA has approved the investigator investigational new drug (IND) application of University of California, Los Angeles (UCLA), the clinical partner of Advanced Cell Technology (ACT), to commence a clinical trial using the human embryonic stem cells (hESCs)-derived cells to treat severe myopia.1 D0 O4 v7 G; P. l

6 x! o0 x6 k8 r' ?Embryonic stem cell-based trial was designed to assess the hESC-derived ACT's retinal pigment epithelial (RPE) cells in patients with severe myopia (nearsightedness).( Q) d8 u0 e* v. G% H# x

. P! w2 D# Z. T) q! I. E1 m' [$ v; YACT chairman and CEO Gary Rabin said, "We are pleased to be on track to broaden the scope of our RPE program with the initiation of the new Investigator IND."
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0 e# U# m+ b* q; {5 v) CAround 12 patients are expected to be enrolled with cohorts of three patients in an ascending dosage design in the myopia trial.( ^5 e7 J4 a; d5 X3 h9 l1 Q
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The primary endpoint of the prospective, open-label study is to assess the safety and tolerability profile of hESC-derived RPE cells subsequent to sub-retinal transplantation into myopia patients at 12 months.
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ACT chief scientific officer Robert Lanza said, "Myopia is one of the most common medical ailments in the world and myopic patients have a higher risk of permanent vision loss due to complications such as fissures in the RPE layer of the eye."