Single atom engineering for radiotherapy-activated immune agonist prodrugs

Abstract Immunotherapy has revolutionized cancer treatment by leveraging the body’s immune system to combat malignancies. However, on-target, off-tumour (OTOT) toxicity poses significant challenges, often leading to the failure of clinical trials for the development of immunotherapeutic drugs. The m...

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Main Authors: Zexuan Ding, Xiaozhe Yin, Yuedan Zheng, Yiyan Li, Huanhuan Ge, Jianshu Feng, Ziyang Wang, Simiao Qiao, Qi Sun, Fashuo Yu, Zhanshan Hou, Yang-Xin Fu, Zhibo Liu
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-60768-4
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author Zexuan Ding
Xiaozhe Yin
Yuedan Zheng
Yiyan Li
Huanhuan Ge
Jianshu Feng
Ziyang Wang
Simiao Qiao
Qi Sun
Fashuo Yu
Zhanshan Hou
Yang-Xin Fu
Zhibo Liu
author_facet Zexuan Ding
Xiaozhe Yin
Yuedan Zheng
Yiyan Li
Huanhuan Ge
Jianshu Feng
Ziyang Wang
Simiao Qiao
Qi Sun
Fashuo Yu
Zhanshan Hou
Yang-Xin Fu
Zhibo Liu
author_sort Zexuan Ding
collection DOAJ
description Abstract Immunotherapy has revolutionized cancer treatment by leveraging the body’s immune system to combat malignancies. However, on-target, off-tumour (OTOT) toxicity poses significant challenges, often leading to the failure of clinical trials for the development of immunotherapeutic drugs. The molecular engineering of clinically relevant, tumour-selective prodrugs, activated in a targeted way, could help minimize systemic toxicity while maximizing anti-tumour efficacy. Here, we propose a Single Atom Engineering for Radiotherapy-Activated Prodrug (SAE-RAP) technique for the development of radiotherapy-activatable small-molecule immune agonist prodrugs. We show that introducing a single oxygen atom into the TLR7/8 agonist R848 significantly reduces the EC50 value by over 4000-fold, hence mitigating severe side effects following systemic administration. In preclinical tumour mouse models, exposure to radiotherapy removes the protective mask provided by the oxygen atom and locally rescues the activity of the prodrugs, triggering anti-tumour immunity and limiting the growth of primary and distal tumours. The SAE-RAP technique may be further utilized for developing radiotherapy-activated prodrugs for next-generation combination therapies that transcend traditional limitations.
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institution Kabale University
issn 2041-1723
language English
publishDate 2025-07-01
publisher Nature Portfolio
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series Nature Communications
spelling doaj-art-ac3be6c7d4bc45efa87f8b75670e6a112025-08-20T04:01:36ZengNature PortfolioNature Communications2041-17232025-07-0116111410.1038/s41467-025-60768-4Single atom engineering for radiotherapy-activated immune agonist prodrugsZexuan Ding0Xiaozhe Yin1Yuedan Zheng2Yiyan Li3Huanhuan Ge4Jianshu Feng5Ziyang Wang6Simiao Qiao7Qi Sun8Fashuo Yu9Zhanshan Hou10Yang-Xin Fu11Zhibo Liu12Changping LaboratoryDepartment of Basic Medical Sciences, School of Medicine, Tsinghua UniversityBeijing National Laboratory for Molecular Sciences, Radiochemistry and Radiation Chemistry Key Laboratory of Fundamental Science, Key Laboratory of Bioorganic Chemistry and Molecular Engineering of Ministry of Education, College of Chemistry and Molecular Engineering, Peking UniversityPeking University-Tsinghua University Centre for Life Sciences, Peking UniversityChangping LaboratoryChangping LaboratoryBeijing National Laboratory for Molecular Sciences, Radiochemistry and Radiation Chemistry Key Laboratory of Fundamental Science, Key Laboratory of Bioorganic Chemistry and Molecular Engineering of Ministry of Education, College of Chemistry and Molecular Engineering, Peking UniversityChangping LaboratoryPeking University-Tsinghua University Centre for Life Sciences, Peking UniversityChangping LaboratoryChangping LaboratoryChangping LaboratoryChangping LaboratoryAbstract Immunotherapy has revolutionized cancer treatment by leveraging the body’s immune system to combat malignancies. However, on-target, off-tumour (OTOT) toxicity poses significant challenges, often leading to the failure of clinical trials for the development of immunotherapeutic drugs. The molecular engineering of clinically relevant, tumour-selective prodrugs, activated in a targeted way, could help minimize systemic toxicity while maximizing anti-tumour efficacy. Here, we propose a Single Atom Engineering for Radiotherapy-Activated Prodrug (SAE-RAP) technique for the development of radiotherapy-activatable small-molecule immune agonist prodrugs. We show that introducing a single oxygen atom into the TLR7/8 agonist R848 significantly reduces the EC50 value by over 4000-fold, hence mitigating severe side effects following systemic administration. In preclinical tumour mouse models, exposure to radiotherapy removes the protective mask provided by the oxygen atom and locally rescues the activity of the prodrugs, triggering anti-tumour immunity and limiting the growth of primary and distal tumours. The SAE-RAP technique may be further utilized for developing radiotherapy-activated prodrugs for next-generation combination therapies that transcend traditional limitations.https://doi.org/10.1038/s41467-025-60768-4
spellingShingle Zexuan Ding
Xiaozhe Yin
Yuedan Zheng
Yiyan Li
Huanhuan Ge
Jianshu Feng
Ziyang Wang
Simiao Qiao
Qi Sun
Fashuo Yu
Zhanshan Hou
Yang-Xin Fu
Zhibo Liu
Single atom engineering for radiotherapy-activated immune agonist prodrugs
Nature Communications
title Single atom engineering for radiotherapy-activated immune agonist prodrugs
title_full Single atom engineering for radiotherapy-activated immune agonist prodrugs
title_fullStr Single atom engineering for radiotherapy-activated immune agonist prodrugs
title_full_unstemmed Single atom engineering for radiotherapy-activated immune agonist prodrugs
title_short Single atom engineering for radiotherapy-activated immune agonist prodrugs
title_sort single atom engineering for radiotherapy activated immune agonist prodrugs
url https://doi.org/10.1038/s41467-025-60768-4
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