Dual inhibition of TNF-α and IL-1β signaling through BIG2 ameliorates osteoarthritis in mouse and primate models

Osteoarthritis (OA), a leading cause of disability, is characterized by chronic low-grade inflammation and metabolic dysregulation in chondrocytes. TNF-α and IL-1β are key inflammatory drivers in OA pathogenesis, but clinical trials targeting them individually have been unsuccessful, highlighting the potential need for dual-targeting strategies. By screening a US Food and Drug Administration–approved drug library, we identified dabigatran, a thrombin inhibitor, as a potent dual suppressor of TNF-α and IL-1β signaling that restored chondrocyte metabolism. In preclinical OA models, including both mice and cynomolgus macaques, dabigatran attenuated cartilage degeneration, alleviated pain, and improved joint function. To elucidate the molecular mechanisms, we performed biochemical copurification coupled with mass spectrometry, identifying brefeldin A–inhibited guanine nucleotide–exchange protein 2 (BIG2) as a previously unknown dabigatran-binding target. Mechanistically, dabigatran binds to BIG2, promoting BIG2-dependent TNFR1 release to dampen TNF-α signaling while enhancing BIG2-mediated IL-1R1 degradation through the autophagy-lysosome pathway to limit IL-1β effects. These findings establish BIG2-mediated receptor trafficking and membrane dynamics as a regulatory node that concurrently inhibits TNF-α and IL-1β signaling, suggesting this pathway as a potential therapeutic strategy for OA.

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Publication Details

Journal
Science Translational Medicine
Published
2026-11-11
DOI
https://doi.org/10.1126/scitranslmed.aec8494
Primary Topic
Osteoarthritis Treatment and Mechanisms
Type
article
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article

Dual inhibition of TNF-α and IL-1β signaling through BIG2 ameliorates osteoarthritis in mouse and primate models

Guodong Sun, Ran M. Schwarzkopf, Irvin C. Oh, Lei Zhang et al.
Science Translational Medicine
Osteoarthritis Treatment and Mechanisms
article

Dual inhibition of TNF-α and IL-1β signaling through BIG2 ameliorates osteoarthritis in mouse and primate models

Guodong Sun, Ran M. Schwarzkopf, Irvin C. Oh, Lei Zhang, Chao Wang, Jin Wo, Ronghan Liu, C.-J. Liu, H.-Y. Zhu, Wenhao Song, 崔亚洲, Wenyu Fu, Daniel Howard Wiznia, Shuya Wang, Yuehong Chen, Xinhui Liu
article en

Abstract

Osteoarthritis (OA), a leading cause of disability, is characterized by chronic low-grade inflammation and metabolic dysregulation in chondrocytes. TNF-α and IL-1β are key inflammatory drivers in OA pathogenesis, but clinical trials targeting them individually have been unsuccessful, highlighting the potential need for dual-targeting strategies. By screening a US Food and Drug Administration–approved drug library, we identified dabigatran, a thrombin inhibitor, as a potent dual suppressor of TNF-α and IL-1β signaling that restored chondrocyte metabolism. In preclinical OA models, including both mice and cynomolgus macaques, dabigatran attenuated cartilage degeneration, alleviated pain, and improved joint function. To elucidate the molecular mechanisms, we performed biochemical copurification coupled with mass spectrometry, identifying brefeldin A–inhibited guanine nucleotide–exchange protein 2 (BIG2) as a previously unknown dabigatran-binding target. Mechanistically, dabigatran binds to BIG2, promoting BIG2-dependent TNFR1 release to dampen TNF-α signaling while enhancing BIG2-mediated IL-1R1 degradation through the autophagy-lysosome pathway to limit IL-1β effects. These findings establish BIG2-mediated receptor trafficking and membrane dynamics as a regulatory node that concurrently inhibits TNF-α and IL-1β signaling, suggesting this pathway as a potential therapeutic strategy for OA.

Science Translational MedicineVol. 18(875)
Jinan University (CN), Yale University (US), First Affiliated Hospital of Jinan University (CN), New York University (US)
Osteoarthritis Treatment and Mechanisms
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