Tang Erting, Hu Yifei, Cao Guoshuai, Asby Nicholas W, Nguyen Duy-Thuc, Aboelella Nada S, Ruiz Hanna, Zhao Yu, Xie Lishi, Chen Xiufen, Bishop Michael R, Riedell Peter A, LaBelle James L, Kline Justin P, Huang Jun
Pritzker School of Molecular Engineering, University of Chicago, Chicago, IL 60637, USA.
Pritzker School of Medicine, University of Chicago, Chicago, IL 60637, USA.
bioRxiv. 2025 May 20:2025.05.13.653878. doi: 10.1101/2025.05.13.653878.
CD19-directed chimeric antigen receptor (CAR) T-cell therapy has significantly advanced the treatment landscape for relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL). However, up to 60% of patients do not achieve a complete response. To uncover determinants of therapeutic efficacy, we analyzed the infusion products of eight r/r DLBCL patients with distinct clinical responses to axicabtagene ciloleucel using single-cell transcriptomics. Compared to patients who exhibited progressive disease, infusion products of complete responders demonstrated enriched signatures of type I interferon (IFN-I) signaling. Based on these findings, we developed a novel strategy to improve CD19-directed CAR T-cell treatment efficacy by incorporating IFN-I as an enhancer during the ex vivo manufacturing process. For both CD28- and 4-1BB-costimulated second-generation CARs, we found that low-strength IFN-I signaling enhanced CAR T-cell cytotoxicity and in vivo efficacy. On the other hand, high-strength IFN-I signaling compromised cell viability and in vivo efficacy. Our low-strength IFN-I signaling approach leverages an existing FDA-approved pharmacologic agent and is compatible with current CAR constructs and manufacturing workflows. Together, our results establish IFN-I as a potent and costimulation-independent enhancer of CAR T-cell efficacy and provide a translationally feasible approach to enhance CAR T-cell therapies for r/r DLBCL.
靶向CD19的嵌合抗原受体(CAR)T细胞疗法显著推动了复发/难治性弥漫性大B细胞淋巴瘤(r/r DLBCL)的治疗进展。然而,高达60%的患者未实现完全缓解。为了揭示治疗效果的决定因素,我们使用单细胞转录组学分析了8例对axi-cabtagene ciloleucel有不同临床反应的r/r DLBCL患者的输注产物。与疾病进展的患者相比,完全缓解者的输注产物显示出I型干扰素(IFN-I)信号的富集特征。基于这些发现,我们开发了一种新策略,通过在体外制造过程中加入IFN-I作为增强剂来提高靶向CD19的CAR T细胞治疗效果。对于CD28共刺激和4-1BB共刺激的第二代CAR,我们发现低强度IFN-I信号增强了CAR T细胞的细胞毒性和体内疗效。另一方面,高强度IFN-I信号损害了细胞活力和体内疗效。我们的低强度IFN-I信号方法利用了一种现有的FDA批准的药物,并且与当前的CAR构建体和制造工作流程兼容。总之,我们的结果确立了IFN-I作为CAR T细胞疗效的一种有效且不依赖共刺激的增强剂,并提供了一种可转化的可行方法来增强r/r DLBCL的CAR T细胞疗法。