基层细胞产品:挑战和临床治疗选择
Debora B Mello1, Fernanda Cristina Paccola Mesquita2, Danúbia Silva Dos Santos3,4
1National Center of Structural Biology and Bioimaging, CENABIO, Federal University of Rio de Janeiro, Rio de Janeiro 21941-902, Brazil.
International journal of molecular sciences
|June 19, 2024
概括
介质细胞 stromal cell (MSC) 疗法看起来有前途,但由于制造变异性而面临挑战. 建立最低制造标准对于推进这些先进治疗药物 (ATMP) 的再生医学至关重要.
科学领域:
- 再生医学是一种再生医学.
- 细胞疗法制造业 细胞疗法制造业
- 生物技术是生物技术.
背景情况:
- 介质细胞 stromal 细胞 (MSCs) 用于先进治疗药物 (ATMPs) 的各种临床应用.
- MSC来源各不相同,包括捐赠组织,诱导多能干细胞和胚胎干细胞.
- 在ATMP的异质性可以影响产品的身份和效力,影响临床结果.
研究的目的:
- 审查与基于介质干细胞 (MSC) 的先进治疗药物 (ATMP) 相关的挑战.
- 突出制造变异对产品标识,效能和临床试验成功的影响.
- 强调需要在再生医学中为MSC标准化制造标准.
主要方法:
- 对MSC隔离和制造当前实践的文献综述.
- 分析导致ATMP异质性的因素.
- 讨论可变性对临床转换的影响.
主要成果:
- 在MSC来源和制造方法中存在显著的变化.
- 产品的异质性对基于MSC的ATMP的身份,功效和有效性构成风险.
- 目前的制造工艺缺乏普遍确定的最低标准.
结论:
- 标准化MSC的制造标准对于确保产品的一致性和治疗效果至关重要.
- 解决制造变异性将提高在临床试验中基于MSC的ATMP的可靠性.
- 建立最低标准将通过强大的基于细胞的疗法促进再生医学的进步.
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