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bioRxiv · 10.1101/2025.10.23.684261

Immunometabolic determinants of long-term response in leukemia patients receiving CD19 CAR T cell therapy

Abstract

Although most patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) receiving CD19-targeted chimeric antigen receptor (CAR) T cell therapy achieve remission, loss of CAR T cell functionality and subsequent relapse remains an unmet therapeutic need. We applied an integrative approach to study the immunometabolism of pre- and post-infusion CD19-CAR T cells of patients with relapsed/refractory B-ALL. Pre-infusion CAR T cells of long-term responders (LTR) had increased oxidative phosphorylation, fatty acid oxidation, and pentose phosphate pathway activities, higher mitochondrial mass, tighter cristae, and lower mTOR expression compared to products of short-term responders. Post-infusion CAR T cells in bone marrow (BM) of LTR had high immunometabolic plasticity and mTOR-pS6 expression supported by the BM microenvironment. Transient inhibition of mTOR during manufacture induced metabolic reprogramming and enhanced anti-tumor activity of CAR T cells. Our findings provide insight into immunometabolic determinants of long-term response and suggest a therapeutic strategy to improve long-term remission.

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BibTeXRIS

Goldberg, L., Haas, E. R., Wu, J., Garcia, B., Urak, R., Vyas, V., Espinosa, R., Munoz, T., Bierkatz, S., PATHAK, K. V., Hansen, N. P., Pirrotte, P., Singhal, J., Figarola, J., Noriega, R. Z., Li, Z., Wi, D., Tanaka, E., Klein Geltink, R. I., Chen, M.-H., Wu, X., Wagner, J. R., Paul, J., Clark, M. C., Ngo, D., Aldoss, I., Forman, S. J., Wang, X.. 2025-10-24. Immunometabolic determinants of long-term response in leukemia patients receiving CD19 CAR T cell therapy. https://doi.org/10.1101/2025.10.23.684261

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