Polycystic ovary syndromes (PCOS) affect 6-18% of reproductive age female, representing the most commonendocrine disease in this population. PCOS causes gynecological, dermatologic and metabolic comorbidities,and also imposes long-term risks of diabetes mellitus type 2 (T2D), cardiovascular diseases (CVD) andpsychiatric problems including severe depression. PCOS is related to complex hormonal dysregulations such ashyperandrogenism, hyperinsulinemia and gonadotropin dysregulation. And PCOS is commonly associated withmetabolic disorders, with 50-70% of PCOS patients showing insulin resistance and 10% with T2D, implying apotential causative interlinks between them. Current standard of care includes life style changes and multiplelines of medications with aims to relieve symptoms. Medications usually have moderate success rates, and havelimited effects on long-term risks of T2D, CVD and psychiatric problems. We propose to use metabolically activebrown adipocytes, which have high secretion of endocrine factors including but not limited to adiponectin, to treatPCOS. The working mechanisms may involve the both metabolic activities and endocrine secretions of brownadipocytes, which are previously extensively studied in metabolic programs and now indicative of clinical benefitfor PCOS treatment as well. The present Phase I SBIR project aims to test the hypothesis based on our bankcurrently containing 150+ cell populations from donor, and evaluate clinically relevant efficacy of the selectedcells in an established mouse model.
Public Health Relevance Statement: Project narrative
PCOS affects 6-18% of reproductive age female with up to 5 million patients in the United States. It is one of
the most common reproductive endocrine diseases. PCOS has a broad set of chronic symptoms related to
hormonal imbalance such as hyperandrogenism, hyperinsulinemia and gonadotropin dysregulation. PCOS
causes menstrual acyclicity, ovarian cysts, anovulation, infertility, hirsutism and acne, and also importantly, the
long-term risks of developing insulin resistance, diabetes mellitus type 2 (T2D), hyperlipidemia, cardiovascular
diseases (CVD) and psychiatric problems including severe depression. We propose to use metabolically active
brown adipocytes, which have high secretion of endocrine factors including but not limited to adiponectin, to
treat PCOS symptoms and resolve long term risks of developing T2D and CVD.
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