对BCG不响应的非肌肉侵入性膀癌的基因介导疗法:机制,临床证据和实际实施
Chris Ho-Ming Wong1, David Ka-Wai Leung1, Paolo Gontero2
1S.H. Ho Urology Centre, Department of Surgery, The Chinese University of Hong Kong, Hong Kong, Hong Kong.
Current opinion in urology
|September 19, 2025
概括
基因疗法为高风险,Bacillus Calmette-Guérin (BCG) 不响应的非肌肉侵入性膀癌 (NMIBC) 提供了一种有前途的膀节约方法. 在分娩和组合疗法的进步正在改善膀的保存和患者的结果.
科学领域:
- 在瘤学瘤学.
- 基因治疗 基因治疗
- 泌尿器科 泌尿器科 泌尿器科 泌尿器科
背景情况:
- 非肌肉侵入性膀癌 (NMIBC) 不对Bacillus Calmette-Guérin (BCG) 治疗有反应,这对治疗提出了重大挑战.
- 对于高风险的NMIBC,经常考虑进行激进囊切除术,但对于符合条件的患者,需要进行膀节省的选择.
- 基因疗法提供了一种新的策略,以解决BCG不响应NMIBC的未满足需求.
研究的目的:
- 审查BCG不响应的非肌肉侵入性膀癌 (NMIBC) 患者基因介导疗法的最新进展.
- 突出基因疗法的潜力,作为膀节约的替代激进囊切除术.
主要方法:
- 在NMIBC中对基因疗法应用的当前文献的审查.
- 分析新兴的基因传递平台和矢量工程.
- 评估与免疫疗法的联合策略.
主要成果:
- 膀的解剖学促进了高效的静脉内基因传递.
- 腺病毒载体,包括FDA批准的纳多法拉基尼 (Adstiladrin) 和瘤性腺病毒 (cretostimogene,CG0070),显示出有希望的结果.
- 与免疫检查点抑制剂的组合疗法增强了基因治疗的耐用性.
- 尿路和血瘤DNA正在成为患者选择和监测的预测生物标志物.
结论:
- 基因介导疗法正在发展成为管理NMIBC的关键组成部分.
- 扩大适应症和强效组合疗法预计将改善膀保存率.
- 基因疗法具有显著的潜力,可以改善NMIBC患者的长期结果.
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