Faculty of Physiology and Pharmacology "V. Erspamer", Sapienza Università di Roma, 00185 Rome, Italy.
National Center for Drug Research and Evaluation, Istituto Superiore di Sanità, 00161 Rome, Italy.
Int J Mol Sci. 2024 Sep 26;25(19):10365. doi: 10.3390/ijms251910365.
Chimeric antigen receptor (CAR) T-cell therapy has achieved notable success in treating hematological cancers but faces significant challenges in solid-tumor treatment and overall efficacy. Key limitations include T-cell exhaustion, tumor relapse, immunosuppressive tumor microenvironments (TME), immunogenicity, and antigen heterogeneity. To address these issues, various genetic engineering strategies have been proposed. Approaches such as overexpression of transcription factors or metabolic armoring and dynamic CAR regulation are being explored to improve CAR T-cell function and safety. Other efforts to improve CAR T-cell efficacy in solid tumors include targeting novel antigens or developing alternative strategies to address antigen diversity. Despite the promising preclinical results of these solutions, challenges remain in translating CAR T-cell therapies to the clinic to enable economically viable access to these transformative medicines. The efficiency and scalability of autologous CAR T-cell therapy production are hindered by traditional, manual processes which are costly, time-consuming, and prone to variability and contamination. These high-cost, time-intensive processes have complex quality-control requirements. Recent advancements suggest that smaller, decentralized solutions such as microbioreactors and automated point-of-care systems could improve production efficiency, reduce costs, and shorten manufacturing timelines, especially when coupled with innovative manufacturing methods such as transposons and lipid nanoparticles. Future advancements may include harmonized consumables and AI-enabled technologies, which promise to streamline manufacturing, reduce costs, and enhance production quality.
嵌合抗原受体 (CAR) T 细胞疗法在治疗血液系统癌症方面取得了显著的成功,但在实体瘤治疗和总体疗效方面仍面临重大挑战。主要的局限性包括 T 细胞耗竭、肿瘤复发、免疫抑制性肿瘤微环境 (TME)、免疫原性和抗原异质性。为了解决这些问题,已经提出了各种基因工程策略。目前正在探索过表达转录因子或代谢装甲和动态 CAR 调控等方法,以提高 CAR T 细胞的功能和安全性。其他旨在提高 CAR T 细胞在实体瘤中的疗效的方法包括靶向新抗原或开发替代策略来解决抗原多样性问题。尽管这些解决方案在临床前取得了有希望的结果,但将 CAR T 细胞疗法转化为临床应用以实现这些变革性药物的经济可行的应用仍然存在挑战。传统的、手动的工艺会阻碍自体 CAR T 细胞疗法的生产效率和可扩展性,这些工艺既昂贵又耗时,而且容易出现变异性和污染。这些高成本、时间密集型的过程需要复杂的质量控制要求。最近的进展表明,微生物反应器和自动化即时护理系统等较小的、分散的解决方案可以提高生产效率、降低成本,并缩短制造时间线,特别是与转座子和脂质纳米颗粒等创新制造方法结合使用时。未来的进展可能包括协调一致的耗材和人工智能技术,这有望简化制造过程、降低成本和提高生产质量。