Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance

Abstract Poly‐ADP‐ribose polymerase (PARP) inhibitors (PARPi) have shown great promise for treating BRCA‐deficient tumors. However, over 40% of BRCA‐deficient patients fail to respond to PARPi. Here, we report that thioparib, a next‐generation PARPi with high affinity against multiple PARPs, includi...

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Main Authors: Li‐Min Wang, Pingyuan Wang, Xiao‐Min Chen, Hui Yang, Shan‐Shan Song, Zilan Song, Li Jia, Hua‐Dong Chen, Xu‐Bin Bao, Ne Guo, Xia‐Juan Huan, Yong Xi, Yan‐Yan Shen, Xin‐Ying Yang, Yi Su, Yi‐Ming Sun, Ying‐Lei Gao, Yi Chen, Jian Ding, Jing‐Yu Lang, Ze‐Hong Miao, Ao Zhang, Jin‐Xue He
Format: Article
Language:English
Published: Springer Nature 2023-01-01
Series:EMBO Molecular Medicine
Subjects:
Online Access:https://doi.org/10.15252/emmm.202216235
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author Li‐Min Wang
Pingyuan Wang
Xiao‐Min Chen
Hui Yang
Shan‐Shan Song
Zilan Song
Li Jia
Hua‐Dong Chen
Xu‐Bin Bao
Ne Guo
Xia‐Juan Huan
Yong Xi
Yan‐Yan Shen
Xin‐Ying Yang
Yi Su
Yi‐Ming Sun
Ying‐Lei Gao
Yi Chen
Jian Ding
Jing‐Yu Lang
Ze‐Hong Miao
Ao Zhang
Jin‐Xue He
author_facet Li‐Min Wang
Pingyuan Wang
Xiao‐Min Chen
Hui Yang
Shan‐Shan Song
Zilan Song
Li Jia
Hua‐Dong Chen
Xu‐Bin Bao
Ne Guo
Xia‐Juan Huan
Yong Xi
Yan‐Yan Shen
Xin‐Ying Yang
Yi Su
Yi‐Ming Sun
Ying‐Lei Gao
Yi Chen
Jian Ding
Jing‐Yu Lang
Ze‐Hong Miao
Ao Zhang
Jin‐Xue He
author_sort Li‐Min Wang
collection DOAJ
description Abstract Poly‐ADP‐ribose polymerase (PARP) inhibitors (PARPi) have shown great promise for treating BRCA‐deficient tumors. However, over 40% of BRCA‐deficient patients fail to respond to PARPi. Here, we report that thioparib, a next‐generation PARPi with high affinity against multiple PARPs, including PARP1, PARP2, and PARP7, displays high antitumor activities against PARPi‐sensitive and ‐resistant cells with homologous recombination (HR) deficiency both in vitro and in vivo. Thioparib treatment elicited PARP1‐dependent DNA damage and replication stress, causing S‐phase arrest and apoptosis. Conversely, thioparib strongly inhibited HR‐mediated DNA repair while increasing RAD51 foci formation. Notably, the on‐target inhibition of PARP7 by thioparib‐activated STING/TBK1‐dependent phosphorylation of STAT1, triggered a strong induction of type I interferons (IFNs), and resulted in tumor growth retardation in an immunocompetent mouse model. However, the inhibitory effect of thioparib on tumor growth was more pronounced in PARP1 knockout mice, suggesting that a specific PARP7 inhibitor, rather than a pan inhibitor such as thioparib, would be more relevant for clinical applications. Finally, genome‐scale CRISPR screening identified PARP1 and MCRS1 as genes capable of modulating thioparib sensitivity. Taken together, thioparib, a next‐generation PARPi acting on both DNA damage response and antitumor immunity, serves as a therapeutic potential for treating hyperactive HR tumors, including those resistant to earlier‐generation PARPi.
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spelling doaj-art-24325773452c46108ba801e5debd9f212025-08-24T11:43:13ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842023-01-0115312410.15252/emmm.202216235Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistanceLi‐Min Wang0Pingyuan Wang1Xiao‐Min Chen2Hui Yang3Shan‐Shan Song4Zilan Song5Li Jia6Hua‐Dong Chen7Xu‐Bin Bao8Ne Guo9Xia‐Juan Huan10Yong Xi11Yan‐Yan Shen12Xin‐Ying Yang13Yi Su14Yi‐Ming Sun15Ying‐Lei Gao16Yi Chen17Jian Ding18Jing‐Yu Lang19Ze‐Hong Miao20Ao Zhang21Jin‐Xue He22State Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesUniversity of Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesPharm‐X Center, School of Pharmacy, Shanghai Jiao Tong UniversityState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesUniversity of Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesState Key Laboratory of Drug Research, Cancer Research Center, Shanghai Institute of Materia Medica, Chinese Academy of SciencesAbstract Poly‐ADP‐ribose polymerase (PARP) inhibitors (PARPi) have shown great promise for treating BRCA‐deficient tumors. However, over 40% of BRCA‐deficient patients fail to respond to PARPi. Here, we report that thioparib, a next‐generation PARPi with high affinity against multiple PARPs, including PARP1, PARP2, and PARP7, displays high antitumor activities against PARPi‐sensitive and ‐resistant cells with homologous recombination (HR) deficiency both in vitro and in vivo. Thioparib treatment elicited PARP1‐dependent DNA damage and replication stress, causing S‐phase arrest and apoptosis. Conversely, thioparib strongly inhibited HR‐mediated DNA repair while increasing RAD51 foci formation. Notably, the on‐target inhibition of PARP7 by thioparib‐activated STING/TBK1‐dependent phosphorylation of STAT1, triggered a strong induction of type I interferons (IFNs), and resulted in tumor growth retardation in an immunocompetent mouse model. However, the inhibitory effect of thioparib on tumor growth was more pronounced in PARP1 knockout mice, suggesting that a specific PARP7 inhibitor, rather than a pan inhibitor such as thioparib, would be more relevant for clinical applications. Finally, genome‐scale CRISPR screening identified PARP1 and MCRS1 as genes capable of modulating thioparib sensitivity. Taken together, thioparib, a next‐generation PARPi acting on both DNA damage response and antitumor immunity, serves as a therapeutic potential for treating hyperactive HR tumors, including those resistant to earlier‐generation PARPi.https://doi.org/10.15252/emmm.202216235homologous recombination repairolaparib‐resistantPARP inhibitorPARP7type I interferons
spellingShingle Li‐Min Wang
Pingyuan Wang
Xiao‐Min Chen
Hui Yang
Shan‐Shan Song
Zilan Song
Li Jia
Hua‐Dong Chen
Xu‐Bin Bao
Ne Guo
Xia‐Juan Huan
Yong Xi
Yan‐Yan Shen
Xin‐Ying Yang
Yi Su
Yi‐Ming Sun
Ying‐Lei Gao
Yi Chen
Jian Ding
Jing‐Yu Lang
Ze‐Hong Miao
Ao Zhang
Jin‐Xue He
Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance
EMBO Molecular Medicine
homologous recombination repair
olaparib‐resistant
PARP inhibitor
PARP7
type I interferons
title Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance
title_full Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance
title_fullStr Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance
title_full_unstemmed Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance
title_short Thioparib inhibits homologous recombination repair, activates the type I IFN response, and overcomes olaparib resistance
title_sort thioparib inhibits homologous recombination repair activates the type i ifn response and overcomes olaparib resistance
topic homologous recombination repair
olaparib‐resistant
PARP inhibitor
PARP7
type I interferons
url https://doi.org/10.15252/emmm.202216235
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