202401 Advancing cures and therapies and ending endometriosis diagnostic delays to improve outcomes (ACT ENDO)

​Program seeks Council approval for an initiative titled: “Advancing cures and therapies and ending endometriosis diagnostic delays to improve outcomes (ACT ENDO)”. Increased research is urgently required to improve our understanding of endometriosis initiation, progression, and pathophysiology as a path toward non-invasive diagnostics, improved treatments, and, ultimately, prevention and cure. The purpose of the ACT ENDO initiative is to provide a platform to support synergistic research programs involving multiple, high-quality projects spanning basic, translational, and human subjects research in the field.

Endometriosis is a chronic, debilitating condition estimated to affect approximately 1 in 10 reproductive-aged women. Endometriosis is associated with symptoms including chronic pelvic pain, dysmenorrhea, dyspareunia, dysuria, dyschezia, and infertility, as well as an increased risk for ovarian cancer, depression, migraines, lupus, multiple sclerosis and other pain and autoimmune conditions. Despite the severe impact of this disorder on quality of life, diagnosis may be delayed up to 10 years due to a normalization of intense menstrual pain by patients and healthcare providers and the requirement for surgical visualization, preferably with histologic confirmation, to make a definitive diagnosis. This delay results in prolonged patient suffering, increased risk for the development of comorbidities, and greater socioeconomic cost. Current medical and surgical treatments carry substantial risks and side effects, are associated with a high rate of recurrence, and are best considered temporizing rather than curative. Non-invasive diagnostics and new treatments have been slow to develop, in part due to the need for better understanding of the pathogenesis and pathophysiology of the disorder. Recent advances in many fields, including genomics, proteomics, stem cell biology, inflammation, cellsignaling, and radiology, are poised to accelerate this research.

This initiative seeks to advance knowledge about endometriosis and accelerate identification of pathways for effective treatment by encouraging partnerships between experts in basic and clinical research to conduct integrated and synergistic research studies that will speed the transfer ofscientific knowledge from bench to bedside and back again.  This initiative will support both the mechanistic and scientific priorities of the branch by enabling transdisciplinary projects in emerging scientific areas, such as the role of stem cells in the pathophysiology of, or treatment for, endometriosis and the application of state-of-the-art omic technologies for novel or improved diagnostics for endometriosis.

Despite increased attention and support from NICHD in recent years, endometriosis is still severely underfunded at NIH, being noted in publications as a top disproportionately underfunded female-specific condition. Endometriosis received only $27M in FY22 compared to, for example, $195M for Inflammatory bowel disease that affects approximately only 1% of US adults.

This proposal directly supports the 2020 NICHD Strategic Plan by addressing an Aspirational goal (“accelerate efforts to definitively diagnose, prevent, and treat endometriosis”), Theme 2 (“Promoting Gynecologic,Andrologic, and Reproductive Health,” and Implementation Objective (“[support research on] progenitor/stem cell regeneration methods for gynecologic and andrologic tissues”).

Program Contact

Candace Tingen
Gynecologic Health and Disease Branch (GHDB)

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