A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma

Abstract Clinical treatment of hepatocellular carcinoma (HCC) with 5-fluorouracil (5-FU), the primary anticancer agent, remains unsatisfactory due to the glutathione (GSH)-associated drug resistance and immunosuppressive microenvironment of HCC. To develop a facile yet robust strategy to overcome 5-...

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Main Authors: Ting Zou, Yun Huang, Zongtao Zhou, Shuangyan He, Jia Liu, Yalan Chen, Hongdu Liu, Zhonghui Luo, Miaoxin Liu, Hua Wei, CuiYun Yu
Format: Article
Language:English
Published: BMC 2024-12-01
Series:Journal of Nanobiotechnology
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Online Access:https://doi.org/10.1186/s12951-024-03027-w
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author Ting Zou
Yun Huang
Zongtao Zhou
Shuangyan He
Jia Liu
Yalan Chen
Hongdu Liu
Zhonghui Luo
Miaoxin Liu
Hua Wei
CuiYun Yu
author_facet Ting Zou
Yun Huang
Zongtao Zhou
Shuangyan He
Jia Liu
Yalan Chen
Hongdu Liu
Zhonghui Luo
Miaoxin Liu
Hua Wei
CuiYun Yu
author_sort Ting Zou
collection DOAJ
description Abstract Clinical treatment of hepatocellular carcinoma (HCC) with 5-fluorouracil (5-FU), the primary anticancer agent, remains unsatisfactory due to the glutathione (GSH)-associated drug resistance and immunosuppressive microenvironment of HCC. To develop a facile yet robust strategy to overcome 5-FU resistance for enhanced immunotherapy treatment of HCC via all dimensional GSH exhaustion, we report in this study construction of a minimalist prodrug consisting of 5-FU linked to an indoleamine-(2,3)-dioxygenase (IDO) inhibitor (IND) via a disulfide bridge, FU-SS-IND that can further self-assemble into stabilized nanoparticles, FU-SS-IND NPs. Specifically, besides the disulfide linker-induced GSH exhaustion, IND inhibits GSH biosynthesis and enhances the effector function of T cells for turning a “cold” tumor to a “hot” one, which synergistically achieving a tumor inhibition rate (TIR) of 92.5% in a 5-FU resistant mice model. Most importantly, FU-SS-IND NPs could upregulate programmed death ligand 1 (PD-L1) expression on the surface of tumor cells, which enables facile combination with immune checkpoint blockade (ICB) for a ultimate prolonged survival lifetime of 5-FU-resistant tumors-bearing mice. Overall, the minimalist bioreducible nano-prodrug developed herein demonstrates great translatable potential for efficiently reversing drug resistance and enhancing immunotherapy of HCC.
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spelling doaj-art-add4d632670f43dc85ef0d3b295690a62025-08-20T02:20:47ZengBMCJournal of Nanobiotechnology1477-31552024-12-0122111810.1186/s12951-024-03027-wA minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinomaTing Zou0Yun Huang1Zongtao Zhou2Shuangyan He3Jia Liu4Yalan Chen5Hongdu Liu6Zhonghui Luo7Miaoxin Liu8Hua Wei9CuiYun Yu10Hunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaHunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science & MOE Key Lab of Rare Pediatric Disease, Hengyang Medical School, University of South ChinaAffiliated Hospital of Hunan Academy of Chinese Medicine, Hunan Academy of Chinese MedicineAbstract Clinical treatment of hepatocellular carcinoma (HCC) with 5-fluorouracil (5-FU), the primary anticancer agent, remains unsatisfactory due to the glutathione (GSH)-associated drug resistance and immunosuppressive microenvironment of HCC. To develop a facile yet robust strategy to overcome 5-FU resistance for enhanced immunotherapy treatment of HCC via all dimensional GSH exhaustion, we report in this study construction of a minimalist prodrug consisting of 5-FU linked to an indoleamine-(2,3)-dioxygenase (IDO) inhibitor (IND) via a disulfide bridge, FU-SS-IND that can further self-assemble into stabilized nanoparticles, FU-SS-IND NPs. Specifically, besides the disulfide linker-induced GSH exhaustion, IND inhibits GSH biosynthesis and enhances the effector function of T cells for turning a “cold” tumor to a “hot” one, which synergistically achieving a tumor inhibition rate (TIR) of 92.5% in a 5-FU resistant mice model. Most importantly, FU-SS-IND NPs could upregulate programmed death ligand 1 (PD-L1) expression on the surface of tumor cells, which enables facile combination with immune checkpoint blockade (ICB) for a ultimate prolonged survival lifetime of 5-FU-resistant tumors-bearing mice. Overall, the minimalist bioreducible nano-prodrug developed herein demonstrates great translatable potential for efficiently reversing drug resistance and enhancing immunotherapy of HCC.https://doi.org/10.1186/s12951-024-03027-w5-FUINDNano-prodrugDrug resistanceGSH exhaustionPD-L1
spellingShingle Ting Zou
Yun Huang
Zongtao Zhou
Shuangyan He
Jia Liu
Yalan Chen
Hongdu Liu
Zhonghui Luo
Miaoxin Liu
Hua Wei
CuiYun Yu
A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma
Journal of Nanobiotechnology
5-FU
IND
Nano-prodrug
Drug resistance
GSH exhaustion
PD-L1
title A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma
title_full A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma
title_fullStr A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma
title_full_unstemmed A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma
title_short A minimalist multifunctional nano-prodrug for drug resistance reverse and integration with PD-L1 mAb for enhanced immunotherapy of hepatocellular carcinoma
title_sort minimalist multifunctional nano prodrug for drug resistance reverse and integration with pd l1 mab for enhanced immunotherapy of hepatocellular carcinoma
topic 5-FU
IND
Nano-prodrug
Drug resistance
GSH exhaustion
PD-L1
url https://doi.org/10.1186/s12951-024-03027-w
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