Stem Cell Transplant and Cellular Therapies Unit, Great Metropolitan Hospital "Bianchi-Melacrino-Morelli", 89133 Reggio Calabria, Italy.
Stem Cell Transplant Program CIC 587, Great Metropolitan Hospital "Bianchi-Melacrino-Morelli", 89133 Reggio Calabria, Italy.
Int J Mol Sci. 2022 Nov 1;23(21):13332. doi: 10.3390/ijms232113332.
The treatment landscape for hematologic malignancies has changed since the recent approval of highly effective chimeric antigen receptor T-cell therapies (CAR-T). Moreover, more than 600 active trials are currently ongoing. However, early enthusiasm should be tempered since several issues are still unsolved and represent the challenges for the coming years. The lack of initial responses and early relapse are some hurdles to be tackled. Moreover, new strategies are needed to increase the safety profile or shorten the manufacturing process during CAR-T cells therapy production. Nowadays, most clinically evaluated CAR-T cells products are derived from autologous immune cells. The use of allogeneic CAR-T cells products generated using cells from healthy donors has the potential to change the scenario and overcome many of these limitations. In addition, CAR-T cells carry a high price tag, and there is an urgent need to understand how to pay for these therapies as many of today's current payment systems do not feature the functionality to address the reimbursement gap. Finally, the clinical experience with CAR-T cells for solid tumors has been less encouraging, and development in this setting is desirable.
自最近批准高效嵌合抗原受体 T 细胞疗法(CAR-T)以来,血液系统恶性肿瘤的治疗格局发生了变化。此外,目前正在进行 600 多项积极的试验。然而,早期的热情应该有所克制,因为仍有一些问题尚未解决,这是未来几年的挑战。缺乏初始反应和早期复发是需要解决的一些障碍。此外,需要新的策略来提高安全性或缩短 CAR-T 细胞治疗生产过程中的制造工艺。如今,大多数临床评估的 CAR-T 细胞产品均源自自体免疫细胞。使用来自健康供体的细胞产生同种异体 CAR-T 细胞产品有可能改变这种情况,并克服许多这些限制。此外,CAR-T 细胞价格高昂,迫切需要了解如何为这些疗法付费,因为许多当前的支付系统没有解决报销缺口的功能。最后,CAR-T 细胞治疗实体瘤的临床经验并不那么令人鼓舞,因此希望在这方面取得进展。