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。
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  g4 M+ l. n3 I$ T6 R" ~FDA approves UCLA IND application to commence embryonic stem cell-based trial7 K5 A3 F- b0 s2 h6 k

- @% P. L3 t8 s1 R8 `$ \4 YPublished 12 February 2013
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The 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.
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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).
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ACT 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."6 W3 n4 O) x+ r
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Around 12 patients are expected to be enrolled with cohorts of three patients in an ascending dosage design in the myopia trial.
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! m" D9 h, W% `# fThe 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.1 Z* ~1 Z0 i- [% i" f1 ?
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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."