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肉瘤的基因与细胞治疗:综述

英文原题:Gene and Cell Therapy for Sarcomas: A Review.

PubMed 2025/03/27(内容时间) Cancers (Basel) Q2 · IF 4.8(JCR 2025)

研究概要

背景:肉瘤的异质性及其由此产生的不同亚型特异性特征、高复发率和远处转移倾向,仍对提供最佳治疗构成重大挑战。

中文摘要

背景:肉瘤具有高度异质性和亚型特异性,复发率高且易发生远处转移,持续给优化治疗带来重大挑战。目的:全面综述肉瘤基因治疗和细胞治疗的现有文献及临床试验。方法:系统检索PubMed、Medline、Google Scholar及ClinicalTrials.gov,检索词包括基因治疗、细胞治疗、NK细胞治疗、CAR-T治疗、病毒疗法、肉瘤和实体瘤等,并手工检索相关文献参考文献;不限语言。临床试验记录其NCT编号、状态、疾病和分期。结果:目前仅有三种肉瘤基因或细胞疗法获政府监管机构批准。Rexin-G是首个靶向所有晚期实体恶性肿瘤(包括化疗难治性骨肉瘤和软组织肉瘤)的肿瘤靶向基因治疗载体,于2007年获菲律宾食品药品监督管理局批准。Gendicine是首个于2003年在中国获批瘤内给药的溶瘤病毒。创新型嵌合抗原受体(CAR)T细胞疗法afami-cel于2024年获美国批准用于滑膜肉瘤。文中还讨论其他有前景的疗法。结论:随着研究进入晚期临床开发阶段,肉瘤基因和细胞疗法前景广阔。将其纳入标准肉瘤治疗方案有望显著改善这类罕见且治疗困难癌症患者的生活质量和临床结局。

展开英文摘要原文

Background : The heterogeneity of sarcomas and resulting distinct sub-type specific characteristics, their high recurrence rates, and tendency for distant metastasis, continue to present significant challenges to providing optimal treatments. Objective : To provide a comprehensive review of current literature and clinical trials in gene and cell therapies for sarcomas. Methods : A comprehensive literature search was conducted utilizing the following databases: PubMed, Medline, Google Scholar and clinicaltrials.gov. Search terms included "gene therapy", "cell therapy", "NK cell therapy, "CAR-T therapy", "virotherapy", "sarcoma", "gene therapy", and "solid tumors". Additional sources were identified through manual searching for references of relevant studies. No language restrictions were set. The NCT number, study status, condition, and phase were noted for clinical trials. Results : There are only three gene and cell therapies for sarcomas that have been approved by a federal regulatory agency. Rexin-G: the first tumor-targeted gene therapy vector designed to target all advanced solid malignancies, including chemo-refractory osteosarcomas and soft tissue sarcomas, was approved by the Philippine FDA in 2007. Gendicine was the first oncolytic virus approved for intratumoral delivery in China in 2003. Afami-cel, an innovative chimeric antigen receptor (CAR) T cell therapy, was approved for synovial sarcoma in the United States in 2024. Other promising therapies are discussed in the text. Conclusions : The future of gene and cell therapy for sarcomas holds great promise, as research moves to late-stage clinical development. The integration of gene and cell therapies into standard sarcoma treatment protocols has the potential to significantly improve the quality of life and outcomes for patients with this rare and challenging group of cancers.

论文信息

作者
Chawla SP、Pang SS、Jain D、Jeffrey S、Chawla NS、Song PY、Hall FL、Gordon EM
单位
Sarcoma Oncology Center/Cancer Center of Southern California, Santa Monica, CA 90403, USA.United States
文献类型
综述
期刊
Cancers2025 Mar 27
原文标识
PubMed 40227707 · DOI 10.3390/cancers17071125