Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston Methodist Hospital, Houston, TX 77030, USA; Department of Bioengineering and Institute for Bioengineering and Biosciences, Instituto Superior Técnico, Universidade de Lisboa, Lisbon, Portugal.
Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston Methodist Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA; Center for Human Immunobiology, Texas Children's Hospital, Houston, TX 77030, USA.
Cell Rep. 2017 Oct 3;21(1):17-26. doi: 10.1016/j.celrep.2017.09.015.
Antigen-independent tonic signaling by chimeric antigen receptors (CARs) can increase differentiation and exhaustion of T cells, limiting their potency. Incorporating 4-1BB costimulation in CARs may enable T cells to resist this functional exhaustion; however, the potential ramifications of tonic 4-1BB signaling in CAR T cells remain unclear. Here, we found that tonic CAR-derived 4-1BB signaling can produce toxicity in T cells via continuous TRAF2-dependent activation of the nuclear factor κB (NF-κB) pathway and augmented FAS-dependent cell death. This mechanism was amplified in a non-self-inactivating gammaretroviral vector through positive feedback on the long terminal repeat (LTR) promoter, further enhancing CAR expression and tonic signaling. Attenuating CAR expression by substitution with a self-inactivating lentiviral vector minimized tonic signaling and improved T cell expansion and anti-tumor function. These studies illuminate the interaction between tonic CAR signaling and the chosen expression platform and identify inhibitory properties of the 4-1BB costimulatory domain that have direct implications for rational CAR design.
嵌合抗原受体(CARs)的非抗原依赖性持续信号转导可增加 T 细胞的分化和耗竭,从而限制其效力。在 CAR 中纳入 4-1BB 共刺激作用可能使 T 细胞能够抵抗这种功能耗竭;然而,CAR T 细胞中持续的 4-1BB 信号转导的潜在后果尚不清楚。在这里,我们发现通过持续的 TRAF2 依赖性核因子 κB(NF-κB)途径激活和增强的 FAS 依赖性细胞死亡,持续的 CAR 衍生的 4-1BB 信号转导可在 T 细胞中产生毒性。该机制通过对长末端重复(LTR)启动子的正反馈在非自我失活的γ逆转录病毒载体中放大,进一步增强了 CAR 的表达和持续信号转导。通过用自我失活的慢病毒载体替代来减弱 CAR 表达,可最大限度地减少持续信号转导,并改善 T 细胞扩增和抗肿瘤功能。这些研究阐明了持续的 CAR 信号转导与所选表达平台之间的相互作用,并确定了 4-1BB 共刺激结构域的抑制特性,这对合理的 CAR 设计具有直接意义。