Endometriosis Research to Develop Non-Invasive Treatment and Diagnosis

Diana W. Bianchi, M.D. Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development

On February 27, a few weeks before the country was gripped by the COVID-19 pandemic, Dr. Tanya Petrossian gave birth to a healthy baby boy.

During her grueling labor, what stunned nurses and doctors was her high pain tolerance.

But Dr. Petrossian is no stranger to pain.

Since adolescence and the onset of menstruation, she has endured severe pain for more than a decade. Doctors gave her conflicting opinions on the cause, from ovarian cancer to appendicitis. She finally was diagnosed and treated on Feb. 27, 2017—3 years to the day before giving birth.

Her diagnosis: Endometriosis. The inflammatory disease is estimated to affect about 10% of reproductive-age women around the world—about 190 million women. Endometriosis is defined by the presence of tissue similar to the lining of the uterus that grows lesions or patches outside the uterus, infecting other parts of the body, including the abdominal and pelvic cavities. In Dr. Petrossian’s case, the disease affected her appendix, colon, and ovaries. The primary symptom of endometriosis is intense pain at the time of menstruation but also unpredictably throughout the cycle, possibly due to the invasion of nerves and/or the release of pain-causing substances into the pelvis. What we don’t understand is why some women have plenty of pain but few endometriosis lesions, while others have lots of lesions, but less pain.

Currently, the only way to definitively diagnose the disorder involves surgery. The hesitation to undergo this invasive procedure, and the lack of appreciation by patients and clinicians of the disorder, has left many women suffering without a diagnosis for up to a decade through adolescence and into young adulthood. Research on the causes and effective therapies for endometriosis is a high priority for the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

NICHD supports endometriosis research through its  Gynecologic Health and Disease Branch and Fertility and Infertility Branch, as well as Division of Intramural Population Health Research (DIPHR) and Division of Intramural Research (DIR).

We funded research that led to the first pill approved by the U.S. Food and Drug Administration specifically to treat pain associated with endometriosis. The medicine, called elagolix (also Orilissa®) is used as part of hormonal therapy, stopping the body’s production of certain hormones to prevent ovulation, menstruation, and the growth of endometriosis.

In addition, ongoing NICHD-supported research from a global collaboration analyzes genetic data obtained from uterine tissue to identify patterns that may help diagnose endometriosis without the need for surgery. One of our grantees, Stacey A. Missmer, Sc.D., Professor of Obstetrics, Gynecology, and Reproductive Biology at Michigan State University is also looking at blood and tissue to detect biomarkers combined with patient characteristics to detect disease types and select successful treatment paths earlier in adolescence.

While we still are looking for the underlying cause, we have gained insights into its progression. Dr. Petrossian’s personal experience with the disorder propelled her to start a small biopharmaceutical company. The company’s lab, supported by an NICHD small business grant, is cultivating tiny molecules that invade and kill endometrial cells at the source, stopping them from spreading and growing painful lesions. This approach is intended to eventually offer a non-hormonal therapeutic. Hormonal therapy, in contrast, pauses endometrial cells from growing but has not been effective for all and can have menopause-like side effects that restrict them to short-term use. The key to Dr. Petrossian’s research is preserving the body’s estrogen, which is helpful to maintain fertility, brain function, and bone strength. Her lab is exploring a different pathway to eliminate existing endometrial lesions and prevent the growth of endometrial cells before they start.

As we continue to navigate women’s health during the challenges presented by COVID-19, we must remember that chronic conditions, including endometriosis, persist. Symptoms may even worsen during this time given heightened stress and diminished access to care.

Overall, reproductive health data and research on menstruation is limited. It is one of the key reasons I have consistently called for protection of women through research rather than from research. Our focus in the next 5 years needs to be increasing our knowledge to better understand symptoms in diverse populations, detect early signs of endometriosis, and non-invasively diagnose and treat the condition.

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