Repositioning GLP-1 Receptor Agonists in Endometrial Cancer: Molecular Rationale, Preclinical Insights, and Translational Opportunities

Endometrial cancer incidence and mortality are rising globally, largely driven by the obesity epidemic. Treatment options remain limited for obesity-associated, hormone-resistant, or fertility-preserving endometrial cancer, highlighting the need for novel therapies that address both tumor biology and metabolic dysfunction. In this study, we synthesize the molecular rationale, preclinical, population-based, and emerging clinical trial evidence on glucagon-like peptide-1 receptor (GLP-1R) agonists (GLP-1 RA) in endometrial cancer. GLP-1R is expressed in benign and malignant endometrial tissues, where activation of cAMP-PKA and AMPK-mTOR signaling has been shown to mediate antiproliferative, proapoptotic, and autophagy-inducing effects. Preclinical models demonstrate that class-wide GLP-1 RAs may restore progesterone receptor expression, overcome hormone resistance, and synergize with progestin therapy; however, the effects may vary by agent. Retrospective studies suggest that combining GLP-1 RAs with local progestin therapy, commonly a levonorgestrel-releasing intrauterine device (preferred in obesity because oral progestin bioavailability is reduced), is associated with a reduced risk of endometrial cancer in high-risk women. Ongoing clinical trials are assessing their role in fertility-sparing settings-evaluating complete response to progestin-based therapy, relapse rates, and time to conception outcomes-as well as in adjuvant settings, in which disease-free survival is a key endpoint; however, gastrointestinal tolerability and the absence of long-term safety data in endometrial cancer populations remain important considerations. As a class, GLP-1 RAs represent a promising therapeutic approach to targeting both the obesogenic milieu and tumor-intrinsic pathways in endometrial cancer; however, agent-specific differences warrant attention. Prospective, subtype-stratified trials are essential to establish their role in comprehensive endometrial cancer care.

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Journal
Clinical Cancer Research
Published
2025-12-02
DOI
https://doi.org/10.1158/1078-0432.ccr-25-2819
Primary Topic
Diabetes Treatment and Management
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article
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article

Repositioning GLP-1 Receptor Agonists in Endometrial Cancer: Molecular Rationale, Preclinical Insights, and Translational Opportunities

Matthew A. Powell, Robert Louis Coleman, Vivek Podder, Priya Singhania et al.
Clinical Cancer Research
Diabetes Treatment and Management
article

Repositioning GLP-1 Receptor Agonists in Endometrial Cancer: Molecular Rationale, Preclinical Insights, and Translational Opportunities

Matthew A. Powell, Robert Louis Coleman, Vivek Podder, Priya Singhania, Andrea R. Hagemann, Brian M. Slomovitz, Thomas J. Herzog
article en

Abstract

Endometrial cancer incidence and mortality are rising globally, largely driven by the obesity epidemic. Treatment options remain limited for obesity-associated, hormone-resistant, or fertility-preserving endometrial cancer, highlighting the need for novel therapies that address both tumor biology and metabolic dysfunction. In this study, we synthesize the molecular rationale, preclinical, population-based, and emerging clinical trial evidence on glucagon-like peptide-1 receptor (GLP-1R) agonists (GLP-1 RA) in endometrial cancer. GLP-1R is expressed in benign and malignant endometrial tissues, where activation of cAMP-PKA and AMPK-mTOR signaling has been shown to mediate antiproliferative, proapoptotic, and autophagy-inducing effects. Preclinical models demonstrate that class-wide GLP-1 RAs may restore progesterone receptor expression, overcome hormone resistance, and synergize with progestin therapy; however, the effects may vary by agent. Retrospective studies suggest that combining GLP-1 RAs with local progestin therapy, commonly a levonorgestrel-releasing intrauterine device (preferred in obesity because oral progestin bioavailability is reduced), is associated with a reduced risk of endometrial cancer in high-risk women. Ongoing clinical trials are assessing their role in fertility-sparing settings-evaluating complete response to progestin-based therapy, relapse rates, and time to conception outcomes-as well as in adjuvant settings, in which disease-free survival is a key endpoint; however, gastrointestinal tolerability and the absence of long-term safety data in endometrial cancer populations remain important considerations. As a class, GLP-1 RAs represent a promising therapeutic approach to targeting both the obesogenic milieu and tumor-intrinsic pathways in endometrial cancer; however, agent-specific differences warrant attention. Prospective, subtype-stratified trials are essential to establish their role in comprehensive endometrial cancer care.

Clinical Cancer ResearchVol. 32(3)
Washington University in St. Louis (US), Texas Oncology (US), Bangladesh Islami University (BD), Mount Sinai Medical Center (US), The US Oncology Network (US), Shaheed Suhrawardy Medical College (BD), University of Cincinnati (US), University of Cincinnati Medical Center (US)
Openalex Percentile: Top 50%
Diabetes Treatment and Management
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