NIH-funded study suggests fenoprofen as a potential therapeutic for disease that affects 1 in 10 U.S. women
WHAT:
Fenoprofen, a nonsteroidal anti-inflammatory drug (NSAID), successfully alleviated pain and inflammation in a rodent model of endometriosis, according to researchers funded by the National Institutes of Health (NIH). They chose the drug after using a computer algorithm to evaluate nearly 1,300 existing compounds for their ability to reverse gene expression related to endometriosis disease. The study was funded by NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
Analysis by researchers at the University of California, San Francisco, using publicly available data from people with endometriosis, returned 299 candidate compounds with seven considered top candidates. These drugs included commonly used treatments for the disease, such as aspirin, as well as those not yet studied for this purpose. The researchers chose fenoprofen for further evaluation because it returned the highest gene expression reversal score and belongs to a drug class—NSAIDS—that is one of the first-line treatments for endometriosis.
Fenoprofen is a prescription drug approved for the relief of mild to moderate pain and is often prescribed for arthritis. The researchers analyzed electronic medical records from five University of California healthcare institutions and found that the drug had been prescribed for less than 1% of patients with endometriosis or related conditions. They then tested fenoprofen in a rodent model of the disease, observing that it successfully alleviated vaginal hyperalgesia, a surrogate marker for endometriosis-related pain.
If future studies in people confirm these findings, the researchers suggest that fenoprofen could be prescribed more frequently to treat endometriosis pain. Their work also supports continued use of their computer-based approach to repurpose other existing drugs as potential therapeutic candidates for endometriosis.
There is an urgent need for new therapies and diagnostics for endometriosis, which occurs when tissue similar to the uterine lining grows outside of the uterus, often causing severe pain and infertility. The disease affects an estimated 10% of U.S. women, yet diagnosis is often delayed. Many existing treatments have challenging side effects, do not treat the source of the disease and leave a chance for recurrence. To help address these issues, NICHD recently launched the Advancing Cures and Therapies and ending ENDOmetriosis diagnostic delays (ACT ENDO) initiative. To learn more about ACT ENDO, visit https://grants.nih.gov/grants/guide/notice-files/NOT-HD-24-016.html.
WHO:
NICHD Director Diana W. Bianchi, M.D., is available to discuss the study findings. To arrange an interview, please e-mail nichdpress@mail.nih.gov or call 301-496-5133.
REFERENCES:
Oskotsky TT et al. Identifying therapeutic candidates for endometriosis through a transcriptomics-based drug repositioning approach. iScience DOI: 10.1016/j.isci.2024.109388 (2024)
The work is from co-first authors Tomiko Oskotsky, M.D., Arohee Bhoja and Daniel Bunis, Ph.D., under the leadership of Linda Giudice, M.D., Ph.D., M.Sc., Stacy McAllister, Ph.D., and Marina Sirota, Ph.D. The project is part of the NICHD-funded Endometriosis Center for Discovery, Innovation, Training, and Community Engagement 
at UCSF.
For more information about NICHD-led advances in endometriosis, visit http://www.nichd.nih.gov/publications/product/529.
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About the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): NICHD leads research and training to understand human development, improve reproductive health, enhance the lives of children and adolescents, and optimize abilities for all. For more information, visit https://www.nichd.nih.gov.
About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit https://www.nih.gov.
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