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【健康资讯】美国批准首个基因疗法

2017-09-03 08:16:08   中国青年网

据新华社华盛顿电(记者林小春)美国政府8月30日批准一种基于改造患者自身免疫细胞的疗法治疗白血病,这是第一种在美国获得批准的基因疗法。专家认为,这开辟了癌症治疗的新篇章。

美国食品和药物管理局当天发表声明说,瑞士诺华公司新疗法已获批准,用于治疗25岁以下复发难治型B细胞急性淋巴细胞白血病患者。这是一个“历史性动作”,将“迎来治疗癌症和其他危及生命的重病的新方式”。

新疗法是近年来发展迅速的一种嵌合抗原受体T细胞(CAR-T)疗法,它先从患者自身采集在免疫反应中发挥重要作用的T细胞,然后重新“编程”,所得T细胞含有嵌合抗原受体,能识别并攻击癌变细胞,因此可重新注入患者体内用于治疗。

一项涉及63名患者的临床试验结果显示,83%的患者在接受新方法治疗3个月后,病情得到缓解。患者接受治疗1年后的复发率为64%,存活率为79%。

美药管局局长斯科特·戈特利布评价说:“有能力重编程病人自身细胞,用它攻击致死性癌症,意味着我们正在进入医学创新的新疆域。诸如基因疗法和细胞疗法这样的新技术拥有变革医学的潜力,成为治疗乃至治愈许多棘手疾病的转折点。”

B细胞急性淋巴细胞白血病是一种难以治疗和容易复发的白血病类型,过去的治疗方案较为有限,儿童和青少年患者5年无复发存活率仅为10%至30%。

诺华公司表示,新疗法是一次性治疗,定价为47.5万美元,但如果第一个月见不到效果将不用患者付费。相比之下,白血病常用疗法骨髓移植在美国第一年的收费介于54万至80万美元之间。

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