细胞和基因治疗中的药物监测:风险管理和长期跟踪的不断变化的挑战
Emile Youssef1, Kari Weddle2, Lisa Zimmerman2
1Kapadi, Inc., 9650 Strickland Road, Raleigh, NC, 27615, USA. emile.youssef@kapadi.com.
Drug safety
|August 9, 2025
概括
细胞和基因疗法提供治疗方法,但存在安全风险. 积极主动,数字增强的药监模型对于长期的患者安全和创新至关重要.
科学领域:
- 先进的治疗方式.
- 再生医学是一种再生医学.
- 基因组医学是一种基因组医学.
背景情况:
- 细胞和基因疗法 (CGT),包括CAR T细胞和CRISPR,正在彻底改变癌症,遗传疾病和神经退行性疾病的治疗方法.
- 虽然CGT具有治疗潜力,但由于长时间的活性,免疫反应和基因组变化,CGT存在重大安全挑战.
研究的目的:
- 审查与CGT相关的毒性,确定安全监测的差距,并提出一个新的药物监测框架.
- 加强患者安全并支持CGT开发和应用的持续创新.
主要方法:
- 综合审查CGT相关的毒性,包括免疫,基因毒性和器官特异性影响.
- 对当前临床试验安全数据采集和长期跟踪基础设施局限性的分析.
- 开发一个风险适应性,数字化支持的药监模型,整合现实数据和AI.
主要成果:
- 确定了免疫并发症,基因毒性和器官特异性影响作为关键的CGT毒性.
- 突出了CGT安全数据收集和全球长期跟踪方面的差距.
- 提出了一个利用数字工具,人工智能和多模式数据进行主动安全监测的药物监测模型.
结论:
- 一个数字化,风险适应的药监系统对于管理CGT安全至关重要.
- 集成的工具,如人工智能,生物传感器和电子患者结果可以改善近乎实时的安全监测.
- 积极的药物监测对于在CGT中平衡创新与长期患者安全和伦理考虑至关重要.
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