在欧盟批准的基于细胞的药物:当前的证据和前景
Stefania Bellino1, Anna La Salvia1, Maria Francesca Cometa1
1National Center for Drug Research and Evaluation, National Institute of Health (Istituto Superiore di Sanità), Rome, Italy.
Frontiers in pharmacology
|August 16, 2023
概括
先进治疗药物 (ATMP) 为罕见疾病和癌症提供了新的希望. 虽然有前途,但制造,成本和安全方面的挑战需要进一步研究以获得更广泛的临床应用.
科学领域:
- 再生医学是一种再生医学.
- 细胞和基因疗法细胞和基因疗法
- 药理学 药理学是指药理学的学科.
背景情况:
- 先进治疗药物 (ATMP) 是使用细胞或基因恢复生物功能的创新治疗方法.
- 它们为尚未满足的医疗需求提供了新的治疗途径,包括罕见疾病,癌症和自身免疫性疾病.
- 自2009年以来,欧盟市场已经批准了24种ATMP.
研究的目的:
- 审查欧盟批准的基于细胞的治疗方法的现有证据.
- 在临床试验和现实环境中评估ATMP的疗效和安全性.
- 确定阻碍ATMP广泛临床应用的局限性.
主要方法:
- 审查欧盟授权的基于细胞的疗法,包括体细胞疗法,组织工程产品和CAR T细胞.
- 从临床试验中获得的疗效和安全数据的分析.
- 对ATMP性能和患者结果的现实证据的评估.
主要成果:
- 对于治疗选择有限的患者来说,ATMPs显示出很大的前景.
- 已批准的疗法包括体细胞疗法,组织工程产品和CAR T细胞.
- 关键的安全问题包括神经毒性和细胞因子释放综合征 (CRS) 与CAR T细胞.
结论:
- 基于细胞的疗法代表了医学上的重大进步.
- 诸如复杂的制造,高成本,产品标记性和安全问题等局限性需要解决.
- 进一步的长期研究和现实世界的证据对于充分了解ATMP的有效性和安全性至关重要.
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