在高危神经母细胞瘤中向GD2的CAR-T细胞:一阶段1/2试验
Franco Locatelli1,2, Daria Pagliara3, Maria A De Ioris3
1Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy. franco.locatelli@opbg.net.
Nature medicine
|August 21, 2025
概括
在患有高危神经母细胞瘤的儿童中,第三代GD2- 基默抗原受体 (CAR) T细胞 (GD2- CART01) 呈现持久缓解. 这种治疗是安全有效的,早期治疗的患者的生存率有所提高.
科学领域:
- 儿童瘤学
- 免疫疗法
- 细胞疗法
背景情况:
- 神经母细胞瘤是一种高风险的儿童癌症,转移性,复发性或耐药性病例的结果不佳.
- 化学抗原受体 (CAR) T细胞疗法为治疗这种侵袭性恶性瘤提供了一个有前途的途径.
研究的目的:
- 介绍GD2- CART01治疗高危神经母细胞瘤儿童的最终安全性和疗效结果.
- 评估整体反应率 (ORR),完全缓解率,整体存活率 (OS) 和GD2- CART01持续性.
主要方法:
- 这一临床试验包括54名患有高风险神经母细胞瘤的儿童.
- 使用第三代GD2-CART01的最大耐受剂量为10 × 10^6个CAR+细胞/公斤.
- 监测安全性,包括免疫效应细胞相关的神经毒性综合征 (ICANS) 和疗效终点.
主要成果:
- 没有新的安全信号;四名儿童使用里米杜西德治疗时出现了ICANS (3级).
- 在临床试验队列中,ORR为66%,在目标人群中为77% (疾病负担较低).
- 试验队列的5年生存率为42. 67%和目标人群为68%;较早的治疗线路观察到更好的生存率.
结论:
- 在高危神经母细胞瘤的儿童中,GD2- CART01治疗是安全的,并诱导持久缓解.
- 早期治疗和较低的疾病负担与更高的生存结果有关.
- 对于耐火性/复发性神经母细胞瘤,GD2- CART01是一个可行的治疗选择.
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