里程碑！西方世界首个干细胞治疗产品Holoclar获欧盟批准

大年初一，正值中国人民欢庆羊年春节之际，欧洲的眼病患者也收获了福音——西方世界首个干细胞治疗产品Holoclar获得欧盟委员会（EC）有条件批准，用于因（物理或化学因素所致）眼部灼伤导致的中度至重度角膜缘干细胞缺乏症（limbal stem-cell deficiency，LSCD）成人患者的治疗。

Holoclar由意大利凯西制药（Chiesi Farmaceutici）研发，是获批的首个含干细胞的先进治疗产品，也是首个用于治疗角膜缘干细胞缺乏症（LSCD）的产品。Holoclar不仅能够作为角膜移植的替代疗法，也可以在大范围眼部损伤的情况下增加角膜移植成功的概率。在临床试验中，Holoclar能够修复眼部角膜损伤，并改善或解决疼痛、畏光等症状，同时可改善患者的视敏度（visual acuity）。

Holoclar是一种活组织产品，类似于一个隐形眼镜，其活性物质为“离体扩增（ex-vivo）的包含干细胞的自体人角膜上皮细胞”，由取自患者角膜未受损区域的一小片活组织制备并在实验室利用细胞培养技术生长而成，可用于替代受损的角膜细胞。其中，角膜缘干细胞负责角膜上皮的连续再生和维持。通过在眼球重建干细胞储备，Holoclar能够启动正常的角膜细胞生长和维持。

Holoclar获批的具体适应症为：由物理或化学眼灼伤导致的单侧或双侧中度至重度角膜缘干细胞缺乏症（LSCD）（定义为：至少两个角膜象限存在浅层角膜新生血管，同时中央角膜受累，严重视力损害）。患者接受Holoclar治疗前，需最少取1-2平方毫米完好的角膜缘（limbus）活组织。另外，Holoclar必须由经过培训的合格医生进行管理，并仅限于医院使用。

膜缘干细胞的缺乏使角膜上皮失去再生和修复的能力，引起角膜结膜上皮化、新生血管长入、慢性炎症、反复上皮缺损、基质瘢痕化以及角膜自溶和溃疡，若不进行治疗，该病最终可导致失明。

英文原文：Europe approves Western world's first stem-cell therapy for rare eye conditionREUTERS - Europe has approved the Western world's first medicine containing stem cells to treat a rare condition caused by burns to the eye, marking a milestone in the use of the technology.

Holoclar, from privately held Italian company Chiesi, was given a marketing green light on Friday by the European Commission for treating so-called limbal stem cell deficiency due to physical or chemical burns. Left untreated, the condition can result in blindness.

The stem-cell therapy is a living-tissue product. It resembles a contact lens and is made from a biopsy taken from a small undamaged area of a patient's cornea and grown in the laboratory using cell culture.

The conditional marketing authorisation had been expected, following a positive recommendation by the European Medicines Agency in December.

Chiesi said the approval meant that Holoclar would be available “in the near future” to all suitable patients in Europe, including people who have suffered eye injuries caused by solvents, acids, abrasive and chemical agents“.

来源：生物谷