Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma

Objective: The high heterogeneity of hepatocellular carcinoma (HCC) renders traditional therapies unable to effectively activate the patient’s immune system to combat tumors. Patients with advanced HCC and T cell functional deficiencies may benefit more from cellular immunotherapy, especially tumor...

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Main Authors: Fang Liu, Hua Chen, Suxin Wu, Chenlu Zhu, Mingji Zhang, Wei Rui, Dong Zhou, Yang Wang, Xin Lin, Xueqiang Zhao, Yunbin Ye
Format: Article
Language:English
Published: China Anti-Cancer Association 2025-04-01
Series:Cancer Biology & Medicine
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Online Access:https://www.cancerbiomed.org/content/22/4/412
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author Fang Liu
Hua Chen
Suxin Wu
Chenlu Zhu
Mingji Zhang
Wei Rui
Dong Zhou
Yang Wang
Xin Lin
Xueqiang Zhao
Yunbin Ye
author_facet Fang Liu
Hua Chen
Suxin Wu
Chenlu Zhu
Mingji Zhang
Wei Rui
Dong Zhou
Yang Wang
Xin Lin
Xueqiang Zhao
Yunbin Ye
author_sort Fang Liu
collection DOAJ
description Objective: The high heterogeneity of hepatocellular carcinoma (HCC) renders traditional therapies unable to effectively activate the patient’s immune system to combat tumors. Patients with advanced HCC and T cell functional deficiencies may benefit more from cellular immunotherapy, especially tumor neoepitope-targeted T cell receptor (TCR)-T cells. Neoepitopes with strong immunogenicity provide precise targets for HCC, further enhancing the efficacy of cellular immunotherapy. Methods: A scalable workflow for identifying neoepitopes from 7 HLA-A*02:01-restricted patients with HCC was established based on whole exome sequencing and bioinformatics analyses, followed by identification of neoepitope-specific TCRs through tetramer-based screening and single-cell TCR cloning technology, which were further validated in the JC4 cell model. The cytotoxicity of CD8+ TCR-T cells was evaluated in neoepitope-positive tumor cell lines or NCG mice. Results: Ten specific neoepitopes were identified, among which neoepitope B and T lymphocyte attenuatorP267L [BTLAP267L (SLNHSVIGL)] exhibited advantageous properties as a potential tumor target. Three TCRs (85-3, 126-5, and 52-3) were confirmed to specifically recognize the neoepitope BTLAP267L, while no cross-recognition of irrelevant or wild-type epitopes was observed. Activated BTLAP267L-specific CD8+ TCR-T cells released extensive perforin, granzyme B, IFN-γ, and TNF-α in vitro , thereby inducing strong cytotoxic effects against BTLAP267L-positive T2 or HCC cell lines. BTLAP267L-specific CD8+ TCR-T cells mediated robust tumor regression due to long-lasting survival and released perforin without causing significant cytotoxic effects on normal organs in murine experiments. Conclusions: This preclinical study demonstrated the beneficial effects of neoepitope BTLAP267L-specific TCR-T cell immunotherapy, unlocking a novel strategy for personalized precision therapy in HCC.
format Article
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institution Kabale University
issn 2095-3941
language English
publishDate 2025-04-01
publisher China Anti-Cancer Association
record_format Article
series Cancer Biology & Medicine
spelling doaj-art-04442d98461c4e9a97ca21f3b4e5c5162025-08-20T03:53:42ZengChina Anti-Cancer AssociationCancer Biology & Medicine2095-39412025-04-0122441243210.20892/j.issn.2095-3941.2024.0434Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinomaFang Liu0Hua Chen1Suxin Wu2Chenlu Zhu3Mingji Zhang4Wei Rui5Dong Zhou6Yang Wang7Xin Lin8Xueqiang Zhao9Yunbin Ye10School of Basic Medical Sciences, Fujian Medical University, Fuzhou 350122, ChinaSchool of Basic Medical Sciences, Tsinghua University, Beijing 100084, ChinaSchool of Basic Medical Sciences, Fujian Medical University, Fuzhou 350122, ChinaSchool of Basic Medical Sciences, Fujian Medical University, Fuzhou 350122, ChinaFujian Key Laboratory of Translational Cancer Medicine, Fuzhou 350014, ChinaSchool of Basic Medical Sciences, Tsinghua University, Beijing 100084, ChinaFujian Key Laboratory of Translational Cancer Medicine, Fuzhou 350014, ChinaLaboratory of Immuno-Oncology, Clinical Oncology School of Fujian Medical University, Fujian Cancer Hospital, Fuzhou 350014, ChinaSchool of Basic Medical Sciences, Tsinghua University, Beijing 100084, ChinaSchool of Basic Medical Sciences, Tsinghua University, Beijing 100084, ChinaLaboratory of Immuno-Oncology, Clinical Oncology School of Fujian Medical University, Fujian Cancer Hospital, Fuzhou 350014, ChinaObjective: The high heterogeneity of hepatocellular carcinoma (HCC) renders traditional therapies unable to effectively activate the patient’s immune system to combat tumors. Patients with advanced HCC and T cell functional deficiencies may benefit more from cellular immunotherapy, especially tumor neoepitope-targeted T cell receptor (TCR)-T cells. Neoepitopes with strong immunogenicity provide precise targets for HCC, further enhancing the efficacy of cellular immunotherapy. Methods: A scalable workflow for identifying neoepitopes from 7 HLA-A*02:01-restricted patients with HCC was established based on whole exome sequencing and bioinformatics analyses, followed by identification of neoepitope-specific TCRs through tetramer-based screening and single-cell TCR cloning technology, which were further validated in the JC4 cell model. The cytotoxicity of CD8+ TCR-T cells was evaluated in neoepitope-positive tumor cell lines or NCG mice. Results: Ten specific neoepitopes were identified, among which neoepitope B and T lymphocyte attenuatorP267L [BTLAP267L (SLNHSVIGL)] exhibited advantageous properties as a potential tumor target. Three TCRs (85-3, 126-5, and 52-3) were confirmed to specifically recognize the neoepitope BTLAP267L, while no cross-recognition of irrelevant or wild-type epitopes was observed. Activated BTLAP267L-specific CD8+ TCR-T cells released extensive perforin, granzyme B, IFN-γ, and TNF-α in vitro , thereby inducing strong cytotoxic effects against BTLAP267L-positive T2 or HCC cell lines. BTLAP267L-specific CD8+ TCR-T cells mediated robust tumor regression due to long-lasting survival and released perforin without causing significant cytotoxic effects on normal organs in murine experiments. Conclusions: This preclinical study demonstrated the beneficial effects of neoepitope BTLAP267L-specific TCR-T cell immunotherapy, unlocking a novel strategy for personalized precision therapy in HCC.https://www.cancerbiomed.org/content/22/4/412neoepitopet cell receptor-t cellimmunotherapyhepatocellular carcinoma
spellingShingle Fang Liu
Hua Chen
Suxin Wu
Chenlu Zhu
Mingji Zhang
Wei Rui
Dong Zhou
Yang Wang
Xin Lin
Xueqiang Zhao
Yunbin Ye
Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
Cancer Biology & Medicine
neoepitope
t cell receptor-t cell
immunotherapy
hepatocellular carcinoma
title Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
title_full Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
title_fullStr Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
title_full_unstemmed Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
title_short Neoepitope BTLAP267L-specific TCR-T cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
title_sort neoepitope btlap267l specific tcr t cell immunotherapy unlocks precision treatment for hepatocellular carcinoma
topic neoepitope
t cell receptor-t cell
immunotherapy
hepatocellular carcinoma
url https://www.cancerbiomed.org/content/22/4/412
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