Honaker Yuchi, Gruber David, Jacobs Chester, Yu-Hong Cheng Rene, Patel Shivani, Galvan Christopher Zavala, Khan Iram F, Zhou Kevin, Sommer Karen, Astrakhan Alexander, Cook Peter J, James Richard G, Rawlings David J
Program for Cell and Gene Therapy and Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA 98101, USA.
2seventy Bio, Seattle, WA 98102, USA.
Mol Ther. 2025 Jun 4;33(6):2819-2833. doi: 10.1016/j.ymthe.2024.12.018. Epub 2024 Dec 12.
Pathogenic long-lived plasma cells (LLPCs) secrete autoreactive antibodies, exacerbating autoimmune diseases and complicating solid organ transplantation. Targeted elimination of the autoreactive B cell pool represents a promising therapeutic strategy, yet current treatment modalities fall short in depleting mature PCs. Here, we demonstrate that chimeric antigen receptor (CAR) T cells, targeting B cell maturation antigen (BCMA) utilizing a split-receptor design, offer a controlled and effective therapeutic strategy against LLPCs. Dimerizing agent-regulated immune-receptor complex (DARIC) T cells demonstrated robust rapamycin-dependent targeting of tumor and PCs. Notably, in humanized mouse models, DARIC T cells regulated peripheral human immunoglobulin levels through specific elimination of human LLPCs from the bone marrow. Furthermore, DARIC constructs were efficiently integrated into the T cell receptor α constant (TRAC) locus while maintaining potent antigen-specific cytotoxicity. These findings underscore the potential of split-receptor CAR T cells in autoimmune and transplant medicine, highlighting their versatility in applications beyond oncology.
致病性长寿浆细胞(LLPCs)分泌自身反应性抗体,加剧自身免疫性疾病并使实体器官移植复杂化。靶向清除自身反应性B细胞库是一种很有前景的治疗策略,但目前的治疗方法在耗尽成熟浆细胞方面存在不足。在此,我们证明,利用分裂受体设计靶向B细胞成熟抗原(BCMA)的嵌合抗原受体(CAR)T细胞,为针对LLPCs提供了一种可控且有效的治疗策略。二聚化剂调节的免疫受体复合物(DARIC)T细胞表现出对肿瘤和浆细胞强大的雷帕霉素依赖性靶向作用。值得注意的是,在人源化小鼠模型中,DARIC T细胞通过从骨髓中特异性清除人LLPCs来调节外周人免疫球蛋白水平。此外,DARIC构建体被有效地整合到T细胞受体α恒定区(TRAC)基因座中,同时保持强大的抗原特异性细胞毒性。这些发现强调了分裂受体CAR T细胞在自身免疫和移植医学中的潜力,突出了它们在肿瘤学以外应用中的多功能性。