Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse

Background Adaptive CD19-targeted chimeric antigen receptor (CAR) T-cell transfer has become a promising treatment for leukemia. Although patient responses vary across different clinical trials, reliable methods to dissect and predict patient responses to novel therapies are currently lacking. Recen...

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Main Authors: Chao Ma, Weiqiang Chen, Lunan Liu, Zhuoyu Zhang, Matthew T Witkowski, Iannis Aifantis, Saba Ghassemi
Format: Article
Language:English
Published: BMJ Publishing Group 2022-12-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/10/12/e005360.full
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author Chao Ma
Weiqiang Chen
Lunan Liu
Zhuoyu Zhang
Matthew T Witkowski
Iannis Aifantis
Saba Ghassemi
author_facet Chao Ma
Weiqiang Chen
Lunan Liu
Zhuoyu Zhang
Matthew T Witkowski
Iannis Aifantis
Saba Ghassemi
author_sort Chao Ma
collection DOAJ
description Background Adaptive CD19-targeted chimeric antigen receptor (CAR) T-cell transfer has become a promising treatment for leukemia. Although patient responses vary across different clinical trials, reliable methods to dissect and predict patient responses to novel therapies are currently lacking. Recently, the depiction of patient responses has been achieved using in silico computational models, with prediction application being limited.Methods We established a computational model of CAR T-cell therapy to recapitulate key cellular mechanisms and dynamics during treatment with responses of continuous remission (CR), non-response (NR), and CD19-positive (CD19+) and CD19-negative (CD19−) relapse. Real-time CAR T-cell and tumor burden data of 209 patients were collected from clinical studies and standardized with unified units in bone marrow. Parameter estimation was conducted using the stochastic approximation expectation maximization algorithm for nonlinear mixed-effect modeling.Results We revealed critical determinants related to patient responses at remission, resistance, and relapse. For CR, NR, and CD19+ relapse, the overall functionality of CAR T-cell led to various outcomes, whereas loss of the CD19+ antigen and the bystander killing effect of CAR T-cells may partly explain the progression of CD19− relapse. Furthermore, we predicted patient responses by combining the peak and accumulated values of CAR T-cells or by inputting early-stage CAR T-cell dynamics. A clinical trial simulation using virtual patient cohorts generated based on real clinical patient datasets was conducted to further validate the prediction.Conclusions Our model dissected the mechanism behind distinct responses of leukemia to CAR T-cell therapy. This patient-based computational immuno-oncology model can predict late responses and may be informative in clinical treatment and management.
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spelling doaj-art-0f7bed6b23064c45b3867d327a93ca032024-11-24T04:40:09ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262022-12-01101210.1136/jitc-2022-005360Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapseChao Ma0Weiqiang Chen1Lunan Liu2Zhuoyu Zhang3Matthew T Witkowski4Iannis Aifantis5Saba Ghassemi6Department of Mechanical and Aerospace Engineering, New York University, Brooklyn, New York, USADepartment of Anesthesiology, Shantou Central Hospital, Shantou, Guangdong, ChinaDepartment of Mechanical and Aerospace Engineering, New York University, Brooklyn, New York, USADepartment of Mechanical and Aerospace Engineering, New York University, Brooklyn, New York, USAPerlmutter Cancer Center, NYU Langone Health, New York City, New York, USAPerlmutter Cancer Center, NYU Langone Health, New York City, New York, USA1Center for Cellular Immunotherapies, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USABackground Adaptive CD19-targeted chimeric antigen receptor (CAR) T-cell transfer has become a promising treatment for leukemia. Although patient responses vary across different clinical trials, reliable methods to dissect and predict patient responses to novel therapies are currently lacking. Recently, the depiction of patient responses has been achieved using in silico computational models, with prediction application being limited.Methods We established a computational model of CAR T-cell therapy to recapitulate key cellular mechanisms and dynamics during treatment with responses of continuous remission (CR), non-response (NR), and CD19-positive (CD19+) and CD19-negative (CD19−) relapse. Real-time CAR T-cell and tumor burden data of 209 patients were collected from clinical studies and standardized with unified units in bone marrow. Parameter estimation was conducted using the stochastic approximation expectation maximization algorithm for nonlinear mixed-effect modeling.Results We revealed critical determinants related to patient responses at remission, resistance, and relapse. For CR, NR, and CD19+ relapse, the overall functionality of CAR T-cell led to various outcomes, whereas loss of the CD19+ antigen and the bystander killing effect of CAR T-cells may partly explain the progression of CD19− relapse. Furthermore, we predicted patient responses by combining the peak and accumulated values of CAR T-cells or by inputting early-stage CAR T-cell dynamics. A clinical trial simulation using virtual patient cohorts generated based on real clinical patient datasets was conducted to further validate the prediction.Conclusions Our model dissected the mechanism behind distinct responses of leukemia to CAR T-cell therapy. This patient-based computational immuno-oncology model can predict late responses and may be informative in clinical treatment and management.https://jitc.bmj.com/content/10/12/e005360.full
spellingShingle Chao Ma
Weiqiang Chen
Lunan Liu
Zhuoyu Zhang
Matthew T Witkowski
Iannis Aifantis
Saba Ghassemi
Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse
Journal for ImmunoTherapy of Cancer
title Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse
title_full Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse
title_fullStr Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse
title_full_unstemmed Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse
title_short Computational model of CAR T-cell immunotherapy dissects and predicts leukemia patient responses at remission, resistance, and relapse
title_sort computational model of car t cell immunotherapy dissects and predicts leukemia patient responses at remission resistance and relapse
url https://jitc.bmj.com/content/10/12/e005360.full
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