药物重新发现协议有助于扩大已有的抗癌药物的使用
D L van der Velden1,2, L R Hoes1,2,3, H van der Wijngaart2,3,4
1Department of Molecular Oncology & Immunology, Netherlands Cancer Institute, Amsterdam, The Netherlands.
在使用非标签药物治疗的34%罕见癌症变异患者中,药物重新发现协议发现了临床益处. 这项精确瘤试验加速了新的癌症治疗见解, 并分享了未来治疗决策的知识.
科学领域:
- 癌症学
- 遗传学
- 药理学
背景情况:
- 大规模的瘤基因分析可以识别可操作的分子变异.
- 没有系统地收集非标签药物使用的成功/失败情况.
- 在精密瘤学中需要系统地收集数据.
研究的目的:
- 启动药物重新发现方案, 一个适应精确瘤学试验.
- 在特定患者群体中确定非标签抗癌药物使用的活性信号.
- 在罕见的癌症小组中促进经批准的药物的标签之外的定义使用.
主要方法:
- 适应性,精确瘤学试验设计.
- 已耗尽/拒绝标准疗法和可行的变种的患者的招募.
- 使用未经批准的标签进行向治疗或免疫治疗.
主要成果:
- 在215名接受治疗的患者中,整体临床益处率为34% (136名向治疗,79名免疫治疗).
- 临床益处的中位持续时间为9个月.
- 鉴定了一组成功的微卫星不稳定瘤治疗nivolumab (63%的益处率).
结论:
- 药物重新发现协议在罕见的癌症小组中促进非标签药物使用.
- 识别活动的早期信号,加速新见解的临床转化.
- 为未来的决策创建一个公共知识库.
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