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.
背景：肉瘤的异质性及其导致的独特亚型特异性、高复发率和远处转移倾向，持续为提供最佳治疗方案带来重大挑战。 目的：系统综述当前肉瘤基因与细胞治疗领域的文献及临床试验进展。 方法：通过PubMed、Medline、Google Scholar及clinicaltrials.gov数据库进行系统性文献检索，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”“实体瘤”等。通过人工追溯相关研究的参考文献补充检索来源，不设语言限制。临床试验记录其NCT编号、研究状态、适应症及研究阶段。 结果：目前仅有三项肉瘤基因与细胞疗法获得国家监管机构批准：Rexin-G（首个靶向所有晚期实体恶性肿瘤的基因治疗载体，涵盖化疗难治性骨肉瘤与软组织肉瘤）于2007年获菲律宾FDA批准；Gendicine（全球首个溶瘤病毒制剂）于2003年获中国批准用于瘤内注射；创新性嵌合抗原受体（CAR）T细胞疗法Afami-cel于2024年在美国获批用于滑膜肉瘤。文中同时探讨了其他具有潜力的治疗方案。 结论：随着研究进入后期临床开发阶段，肉瘤基因与细胞治疗前景广阔。将此类疗法整合至标准肉瘤治疗方案中，有望显著改善这类罕见且难治性癌症患者的生活质量与临床结局。
肿瘤(癌症)患者之家