Targeting an atypical G protein–coupled receptor signaling pathway for cardiac fibrosis therapy

Cardiac fibrosis independently predicts adverse outcomes in heart failure (HF), yet no Food and Drug Administration–approved therapy directly targets fibrotic remodeling in the heart. To address this unmet clinical need, we used a multidimensional drug discovery pipeline centered on a human induced pluripotent stem cell (iPSC)–based platform. Through high-throughput screening, we identified CGS15943 (CGS) as the lead antifibrotic compound and validated its activity in human cardiac fibroblasts, three-dimensional engineered heart tissues, and animal models of HF. Mechanistic studies revealed an atypical adenosine receptor (AR)–dependent signaling pathway in which AR subtypes converge on Gβγ (the βγ subunits of heterotrimeric GTP-binding proteins) to activate phosphoinositide 3-kinase (PI3K)–AKT and yes-associated protein (YAP). CGS suppressed this signaling axis, thereby reducing fibrotic gene expression and fibroblast activation. These findings establish AR-driven Gβγ signaling as a potential therapeutic target for cardiac fibrosis.

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Publication Details

Journal
Science
Published
2026-09-24
DOI
https://doi.org/10.1126/science.aej5896
Primary Topic
Cardiac Fibrosis and Remodeling
Type
article
Field-Weighted Citation Impact
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article

Targeting an atypical G protein–coupled receptor signaling pathway for cardiac fibrosis therapy

Remi Janicot, Mark Chandy, Dirk H. Siepe, Danielle H. Shin et al.
Science
Cardiac Fibrosis and Remodeling
article

Targeting an atypical G protein–coupled receptor signaling pathway for cardiac fibrosis therapy

Remi Janicot, Mark Chandy, Dirk H. Siepe, Danielle H. Shin, Nipavan Chiamvimonvat, Marcin Maziarz, Lu Ren, Joseph C. Wu, Phung Ngoc Thai, Jonathan C. Deutsch, Rabindra V. Shivnaraine, Hao Zhang, Wenjuan Zhu, Chengyi Tu, Hyeonyu Kim, Mikel Garcia‐Marcos, Rihua Huang, Brian K. Kobilka, Marian Kalocsay, Chun Liu, Wenqiang Liu, Ying Liu
article en

Abstract

Cardiac fibrosis independently predicts adverse outcomes in heart failure (HF), yet no Food and Drug Administration–approved therapy directly targets fibrotic remodeling in the heart. To address this unmet clinical need, we used a multidimensional drug discovery pipeline centered on a human induced pluripotent stem cell (iPSC)–based platform. Through high-throughput screening, we identified CGS15943 (CGS) as the lead antifibrotic compound and validated its activity in human cardiac fibroblasts, three-dimensional engineered heart tissues, and animal models of HF. Mechanistic studies revealed an atypical adenosine receptor (AR)–dependent signaling pathway in which AR subtypes converge on Gβγ (the βγ subunits of heterotrimeric GTP-binding proteins) to activate phosphoinositide 3-kinase (PI3K)–AKT and yes-associated protein (YAP). CGS suppressed this signaling axis, thereby reducing fibrotic gene expression and fibroblast activation. These findings establish AR-driven Gβγ signaling as a potential therapeutic target for cardiac fibrosis.

ScienceVol. 393(6818)
Boston University (US), Northeastern University (US), The University of Texas MD Anderson Cancer Center (US), University of California, Los Angeles (US), University of Phoenix (US), Medical College of Wisconsin (US), Cardiovascular Institute of the South (US), University of Massachusetts Boston (US), Stanford Cardiovascular Institute (US), University of California, Davis (US), The University of Texas Southwestern Medical Center (US), Stanford University (US)
Good health and well-being
Openalex Percentile: Top 11%
Cardiac Fibrosis and Remodeling
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