Department of Pathology and.
Graduate Program in Immunology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
J Clin Invest. 2024 Jan 2;134(1):e171621. doi: 10.1172/JCI171621.
Even with the prolific clinical use of next-generation cancer therapeutics, many tumors remain unresponsive or become refractory to therapy, creating a medical need. In cancer, DCs are indispensable for T cell activation, so there is a restriction on cytotoxic T cell immunity if DCs are not present in sufficient numbers in the tumor and draining lymph nodes to take up and present relevant cancer antigens. To address this bottleneck, we developed a therapeutic based on albumin fused with FMS-related tyrosine kinase 3 ligand (Alb-Flt3L) that demonstrated superior pharmacokinetic properties compared with Flt3L, including significantly longer half-life, accumulation in tumors and lymph nodes, and cross-presenting-DC expansion following a single injection. We demonstrated that Alb-Flt3L, in combination with standard-of-care chemotherapy and radiation therapy, serves as an in situ vaccination strategy capable of engendering polyclonal tumor neoantigen-specific immunity spontaneously. In addition, Alb-Flt3L-mediated tumor control synergized with immune checkpoint blockade delivered as anti-PD-L1. The mechanism of action of Alb-Flt3L treatment revealed a dependency on Batf3, type I IFNs, and plasmacytoid DCs. Finally, the ability of Alb-Flt3L to expand human DCs was explored in humanized mice. We observed significant expansion of human cross-presenting-DC subsets, supporting the notion that Alb-Flt3L could be used clinically to modulate human DC populations in future cancer therapeutic regimens.
即使新一代癌症治疗药物在临床上得到广泛应用,许多肿瘤仍然对治疗无反应或产生耐药性,这就产生了医疗需求。在癌症中,树突状细胞对于 T 细胞的激活是不可或缺的,因此,如果肿瘤和引流淋巴结中没有足够数量的树突状细胞来摄取和呈递相关的癌症抗原,细胞毒性 T 细胞的免疫就会受到限制。为了解决这个瓶颈问题,我们开发了一种基于白蛋白融合 FMS 相关酪氨酸激酶 3 配体(Alb-Flt3L)的治疗方法,与 Flt3L 相比,它具有更好的药代动力学特性,包括更长的半衰期、在肿瘤和淋巴结中的积累以及单次注射后的交叉呈递-DC 扩增。我们证明,Alb-Flt3L 与标准护理化疗和放射治疗联合使用,是一种能够自发产生多克隆肿瘤新抗原特异性免疫的原位疫苗策略。此外,Alb-Flt3L 介导的肿瘤控制与免疫检查点阻断协同作用,即抗 PD-L1。Alb-Flt3L 治疗的作用机制揭示了对 Batf3、I 型干扰素和浆细胞样树突状细胞的依赖性。最后,在人源化小鼠中探索了 Alb-Flt3L 扩增人树突状细胞的能力。我们观察到人类交叉呈递-DC 亚群的显著扩增,支持 Alb-Flt3L 可用于临床调节未来癌症治疗方案中的人类树突状细胞群体的观点。