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CAR-T 细胞疗法生产的经济有效策略

英文原题:Cost-effective strategies for CAR-T cell therapy manufacturing.

查看英文原题

Cost-effective strategies for CAR-T cell therapy manufacturing.

PubMed 2025/04/03(内容时间) Mol Ther Oncol Q1 · IF 8.5(JCR 2025)

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中文摘要

CAR-T 细胞疗法已改变癌症治疗,获批适应证包括急性B细胞白血病、大B细胞淋巴瘤(LBCL)、滤泡性淋巴瘤(FL)、套细胞淋巴瘤(MCL)和多发性骨髓瘤。

然而,高昂成本限制了疗法可及性。造成成本较高的主要因素包括个体化自体治疗需求、需将患者转运至专科中心、依赖需高级实验室的病毒载体,以及细胞扩增周期长。为解决这些挑战,替代策略旨在简化生产流程并降低复杂性。采用睡美人、piggyBac和CRISPR等非病毒载体,并通过纳米颗粒或电穿孔递送,是有前景的解决方案。这些方法可简化制造、免除病毒载体需求并降低相关成本。

此外,缩短细胞扩增时间和优化方案可显著加快生产。另一新兴方法是使用健康供者经基因编辑的T细胞,制备可治疗多名患者的通用CAR-T 产品。

最后,分散式床旁(POC)CAR-T 制备可最大限度降低物流开支、免除复杂基础设施需求,并使生产地点更接近患者。这一创新策略有望扩大疗法可及性并降低成本,推动CAR-T 疗法更加普及。上述进展结合起来,有望使这一突破性治疗更适用于全球卫生系统。

展开英文摘要原文

CAR-T cell therapy has revolutionized cancer treatment, with approvals for conditions like acute B-leukemia, large B cell lymphoma (LBCL), follicular lymphoma (FL), mantle cell lymphoma (MCL), and multiple myeloma.

However, its high costs limit accessibility. Key factors driving these costs include the need for personalized, autologous treatments, transportation to specialized facilities, reliance on viral vectors requiring advanced laboratories, and lengthy cell expansion processes.

To address these challenges, alternative strategies aim to simplify and reduce production complexity. Non-viral vectors, such as Sleeping Beauty, piggyBac, and CRISPR, delivered via nanoparticles or electroporation, present promising solutions. These methods could streamline manufacturing, eliminate the need for viral vectors, and reduce associated costs.

Furthermore, shortening cell expansion periods and optimizing protocols could significantly accelerate production. An emerging approach involves using genetically edited T cells from healthy donors to create universal CAR-T products capable of treating multiple patients.

Finally, decentralized point-of-care (POC) manufacturing of CAR-T cells minimize logistical expenses, eliminating the need for complex infrastructure, and enabling localized production closer to patients. This innovative strategy holds potential for broadening access and reducing costs, representing a step toward democratizing CAR-T therapy. Combined, these advances could make this groundbreaking treatment more feasible for healthcare systems worldwide.

论文信息

作者
Abdo L、Batista-Silva LR、Bonamino MH
单位
Cell and Gene Therapy Program, Research Coordination, National Cancer Institute (INCA), Rio de Janeiro 20231-050, Brazil.Brazil
文献类型
综述
期刊
Molecular therapy. Oncology2025 Jun 18
原文标识
PubMed 40291594 · DOI 10.1016/j.omton.2025.200980