Treating endometriosis by restoring signaling defects in the endometrial stem cell niche

Funding Type: 
New Faculty Physician Scientist
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

This research will investigate whether the stem cells in a woman’s uterine lining cause endometriosis. Endometriosis is a chronic disease where tissue that normally lines the uterus grows in other parts of a woman’s body causing pain and discomfort. It typically begins around age 25 and lasts until menopause. Up to 10% of women are affected by endometriosis. It is a leading cause of infertility. There is no cure and treatments are often ineffective. The researchers believe that endometriosis may be caused by a miscommunication between one type of stem cell, the ‘uterine epithelial stem cell’, and its environment. This miscommunication may cause the cell to stop responding normally to progesterone, a hormone involved in a woman’s menstrual cycle. The researchers aim to test this idea by comparing the stem cells of women who have endometriosis with the stem cells of women who do not. They will look for differences in cell activity levels and in receptivity to progesterone. If reduced progesterone receptivity is found, they will repair the cells’ progesterone receptors and see if this prevents endometriosis. The final stage of the research will test medicine that can treat the disease. If it caused by reduced receptivity to progesterone, they will test existing FDA-approved drugs, known as histone deacetylase inhibitors to restore normal progesterone response. If it is caused by higher stem cell activity, they will test drugs to block this activity.

Statement of Benefit to California: 

Endometriosis affects 1.8 million Californian women for an average of 25 years each. When the number of affected individuals is multiplied by the duration of disease it yields the following alarming statistic: women in California suffer from endometriosis for a total of 43 million years (data extracted from the Center for Disease Control and the US Census 2010). By this measure, only arthritis and type 1 diabetes are worse chronic non-lethal diseases. Endometriosis affects women in their most productive years. It causes great pain, infertility and significantly increases their risk of developing migraines, anxiety and depression. The total cost of endometriosis to California is $25 billion a year: $20.1 billion is lost in productivity and $4.7 billion in medical care costs. Eleven hours of productivity is lost per women per week (Nnoaham et al., 2011) resulting in 972 million lost work hours a year in California. Treatment costs $2800 per woman per year (Simoens et al., 2007). This does not include treatments for secondary effects of endometriosis such as migraine. Developing a treatment based on this proposal will have the following benefits to California: 1) relief from the third worst chronic disease in California, 2) savings of billions of dollars from reduced care costs, 3) major revenues from regained productivity and 4) revenue from potential new technology and patents owned by the State and licensed to Californian companies.