Erin B. Cole, Sara Lamcaj, Agnes V. Sydenstricker, Adilyn Voss, Christopher R. Hiner, Hong Hur, Manoj Kandpal, Natalia Valderrama Pena, Jian Hua Zheng, Ying Xiong, Zhongyu Zhu, Cheng Cheng Zhang, Niraj Shrestha, Boro Dropulić, Hing C. Wong, Harris Goldstein
Functional persistence of chimeric antigen receptor T cells (CAR T cells) is limited by conventional CAR T cell manufacturing using anti-CD3/CD28 (αCD3/28) stimulation, which generates terminally differentiated and shorter-lived CAR T cells. We demonstrated that HCW9206, a unique protein scaffold linking interleukin-7 (IL-7), an IL-15/IL-15 receptor α (IL-15Rα) complex, and IL-21, generates CAR T cells without requiring αCD3/28 activation, which are highly enriched in long-lived T memory stem cells (T SCM cells) (>50%) and display potent activity across distinct disease models, HIV-1 or B cell leukemia. In a humanized mouse HIV infection model, HCW9206-generated anti-HIV duoCAR T cells suppressed viremia more effectively than αCD3/28-generated anti-HIV duoCAR T cells. In a xenograft leukemia mouse model, a recall proliferative response and complete clearance of leukemia rechallenge were displayed by HCW9206-generated but not by αCD3/28-generated anti-CD19 CAR T cells. HCW9206, a first-in-class cytokine scaffold–based platform, enables production of more potent CAR T cell–based immunotherapies by generating a CAR T cell population, which is highly functional and also markedly enriched for long-lived T SCM cells. This strategy is broadly applicable to increase persistence and functionality of CAR T cells, enhancing their efficacy for treating infectious disease and cancer.