Girls Just Wanna Have an Endometriosis Drug

Endometriosis affects approximately 10% of girls and women of reproductive age, but treatment and diagnostic options remain limited. This may soon change.

Endometriosis is a condition in which tissue similar to the lining of the uterus grows somewhere else. The disease can cause severe pain in the pelvis, heavy periods, and make it harder to get pregnant.

The condition is often debilitating and places a heavy economic burden on healthcare systems: in the United States, the direct and indirect costs associated with endometriosis range between $78 to 119 billion annually.

Despite its wide prevalence, endometriosis still has no cure, with treatments focusing on symptom management. As the disease is often confused for irritable bowel syndrome, spastic colon, and other conditions, it takes an average of 10 years for patients to receive a proper diagnosis.

Even after being diagnosed, many women face a lack of evidence-based information. A survey in Australia found that more than half of endometriosis patients were advised to become pregnant to manage the condition, with nearly 90% of the reported advice coming from healthcare professionals.

On International Women's Day, we look into the most promising endometriosis treatments and diagnostic tools.

Promise in endometriosis pain management

Last year, researchers from the United Kingdom started a clinical trial called EPIC2, exploring dichloroacetate -— a drug previously used to treat rare metabolic disorders in children — for endometriosis pain management.

The trial, enrolling 100 women with the condition, is based on previous research that discovered that cells from the pelvic wall of women with endometriosis produce higher amounts of lactate, a chemical produced when the cells break down carbohydrates for energy.

Increased lactate creates an environment for endometriosis to develop and grow.

Treating endometriosis cells with dichloroacetate decreased lactate production to normal levels and reduced the size of the endometriosis lesions.

In the ongoing trial, the dose of dichloroacetate for each participant will be determined by which version of a gene called GSTZ1 they carry, which is responsible for metabolizing dichloroacetate in the body.

"This will be the first non-hormonal treatment for endometriosis and has the potential to be a real breakthrough in the management of endometriosis. We are very excited to collaborate with Edinburgh University in the EPIC2 trial," Dr. Lucky Saraswat, Honorary Senior Lecturer at the University of Aberdeen, said in a statement.

Treating endometriosis with antibiotics

A 2023 study published in the Science Translational Medicine provided new insights into the poorly understood mechanisms underlying the condition.

The study discovered that 64% of patients with endometriosis had Fusobacterium in the endometrium — the layer of tissue lining the uterus — compared to only 7% of women in the control group.

If it is proven that Fusobacterium, a bacterium found in gum disease, plays a role in the growth of endometriosis lesions, the discovery may open the door to treating the condition with existing antibiotics.

In the study, antibiotic treatment largely prevented the establishment of endometriosis and reduced the number and weight of endometriotic lesions in the mouse model. However, the findings in mice may not necessarily apply to humans.

Better diagnostics may be coming soon

A surgery called laparoscopy is the only way to confirm endometriosis, partly explaining the long road towards the diagnosis. The procedure involves making a small cut in the abdomen and examining internal organs with a thin, lighted tube that has a video camera.

Healthcare providers may also use ultrasound and magnetic resonance imaging tests, but they do not help to detect small endometriosis lesions or adhesions.

France-based biotech company Ziwig has an ambitious plan to speed up the diagnosis process with Endotest, a new device that examines saliva to look for multiple endometriosis biomarkers.

According to interim data published in the New England Journal of Medicine, Endotest showed sensitivity and specificity of over 95%. Last autumn, Ziwig launched a large-scale, 34-month trial involving 2,200 patients from 10 countries to confirm the results.

In January 2024, French health authorities allowed early access to Endotest, saying it could reduce the number of "unnecessary laparoscopies" carried out in adult patients.

According to the company's website, the test, with a price tag of approximately €800, is already available in 14 European countries and is soon coming to the United States and Canada.

Breakthrough endometriosis treatment may be just around the corner, giving hope to millions of women suffering from the debilitating condition.