Single-cell foundation models predict durable CAR T response despite imperfect cell annotation
Leo Shen, Zhiliang BaiiD, Mingyu YangiD, Na LiiD, Rong FaniD
Abstract
CD19-targeted chimeric antigen receptor (CAR) T cell therapy achieves high initial response rates in B-cell acute lymphoblastic leukemia (B-ALL), yet half of patients relapse within one year. Pre-infusion product composition decoded by single-cell RNA sequencing (scRNA-seq) carries information predictive of long-term CAR T persistence, but extracting this information from individual patients typically requires highly sophisticated bioinformatics expert annotation, limiting clinical translation. Here, we evaluate whether single-cell foundation models (scFMs) can extract clinically actionable information from engineered CAR T products. We applied four scFMs (scGPT, scFoundation, CellPLM and UCE), including fine-tuned versions of scGPT and scFoundation, to paired basal and CD19-stimulated pre-infusion CAR T products from 33 pediatric patients with B-ALL. Although annotation accuracy declined relative to healthy peripheral blood references, scFM-derived cell composition stratified patients with long-duration B-cell aplasia with a leave-one-out cross-validated area under the receiver operating characteristic curve of 0.879 (95% confidence interval, 0.742-0.986). Notably, foundation-model-identified cell proportion analysis matched or exceeded expert annotations for several predictive features, demonstrating that accurate clinical prediction may not require perfect per-cell annotation to begin with. CD8+XCL1/2+ cells were further identified as the biomarker consistently associated with durable CAR T persistence across models under CD19 stimulation, whereas other candidate populations showed limited reproducibility. Finally, we translate these findings into a locally deployable decision-support AI agent that predicts the probability of sustained CAR T persistence from pre-infusion CAR T scRNA-seq data.
02 /Specification & Classification
Click any tag to browse matching papers- Citations
- 0
- Organism
- Assay
- Research Focus
- Approach
Reproducibility ledger
Nobody has reported trying to reproduce this paper yet. Did you replicate an experiment or run the code? Your report saves the next lab a week.
Sign in to file a reproducibility report.
Related papers
- Single-cell antigen-specific activation landscape of CAR T infusion product identifies determinants of CD19 positive relapse in patients with ALL
Z. Bai, S. Woodhouse, … R. Fan·bioRxiv·Apr 15, 2021
- CAR T cell foundation model predicts immunotherapy response
- CART-GPT: A T Cell-Informed AI Linguistic Framework for Interpreting Neurotoxicity and Therapeutic Outcomes in CAR-T Therapy
Tiantian Mao, Xiaojian Shao, … Guangxu Jin·bioRxiv·Aug 8, 2025
- Single-cell profiling of CAR-T CD19 cell phenotypes and immune system dynamics in pediatric BCP-ALL
Aleksandra Oszer, Bartłomiej Pawlik, … Wojciech Michal Mlynarski·bioRxiv·Mar 27, 2025
- CancerFoundation: A single-cell RNA sequencing foundation model to decipher drug resistance in cancer
Alexander Theus, Florian Barkmann, … Valentina Boeva·bioRxiv·Nov 1, 2024
- Empirical Evaluation of Single-Cell Foundation Models for Predicting Cancer Outcomes
Ahmed Roman, Shreya Johri, … Haitham Elmarakeby·bioRxiv·Oct 31, 2025