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。 / N/ V' q5 `' V/ F, e$ m# O: O; c/ w: Z9 ^9 E
FDA approves UCLA IND application to commence embryonic stem cell-based trial F: A5 g) v) z- O8 k6 p J& t2 ]! u% i- K% `. u" G
Published 12 February 20131 f+ ^6 U$ J8 A5 j$ u. K
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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. $ x8 r/ R0 W' M, K7 a- ~( Z0 H 4 y9 x$ `' Z7 ~) X, Q. |) lEmbryonic stem cell-based trial was designed to assess the hESC-derived ACT's retinal pigment epithelial (RPE) cells in patients with severe myopia (nearsightedness). 7 }8 O& |/ N. w+ K! B9 P4 X0 X- ?6 A, a# n5 H6 L
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." / k! b; y6 k' s, R$ ?; f" H6 Y( U; K6 T9 f
Around 12 patients are expected to be enrolled with cohorts of three patients in an ascending dosage design in the myopia trial. 9 V' Y- j7 _( N G8 D& m3 s( E0 F* b4 h! n8 {" R$ @9 E6 J
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. $ G- q* C7 {+ v1 S9 I" l# k! C6 c H
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."