The first endometriosis drug in four decades is on the horizon

The uterus is lined with endometrium, a layer of tissue that thickens during the menstrual cycle. If a fertilized egg does not implant, the lining thins and is shed as a period. However, if endometrial tissue grows abnormally outside the uterus, it can cause destruction. In extreme cases of endometriosis, adhesions can “bind” a woman’s organs together — from ovaries to bladder to intestines — and freeze them in place. Mild cases include severe pain, heavy menstruation, swelling and internal bleeding, fatigue and scar tissue. There is no known cure for infertility, and treatment usually focuses on controlling symptoms through some combination of hormonal birth control, pain relief, or surgery.

The World Health Organization estimates that endometriosis affects one in ten women during their lifetime – approximately equal to the proportion of the global population suffering from diabetes. But while doctors understand why diabetes occurs and how to treat it, their understanding of endometriosis is “30 to 40 years” behind, according to Andrew Horne, professor and president-elect of gynecology and reproductive sciences at the University of Edinburgh. Is. The World Endometriosis Society blames this on lack of research and awareness due to lack of funding.

Things have started changing. A clinical trial of the first non-hormonal, non-surgical treatment for endometriosis, starting in Scotland in 2023, is showing promising results. Dr. Horn says the trial, which he co-leads, came about after taking a closer look at how endometriosis lesions form. By taking samples from patients during diagnostic laparoscopy, his team found that people suffering from peritoneal endometriosis – meaning disease on the lining of the pelvic cavity, which represents about 80% of cases – had higher levels of a chemical called lactate in their pelvis than those with. was much higher than. Without.

Lactate is produced when the body breaks down glucose (and it also causes the uncomfortable stitches that sudden runners can get). The researchers believed that its increased presence indicated a role in the development of endometriosis lesions, possibly similar to lactate’s role in helping cancer cells grow. Scientists then searched for a drug that had already been tested in cancer patients, ultimately settling on dichloroacetate (DCA). It is also used to treat a rare type of metabolic disorder in children in which excess lactic acid builds up in the blood.

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A small group of human patients treated with DCA reported reduced pain and improved quality of life. A trial with a larger group and a placebo arm is next. If the drug is approved, which is likely within the next five to seven years, DCA will be the first new endometriosis treatment discovered in four decades.

“There’s still an issue — and I hate to say it — with issues that only affect women,” says Dr. Horn. “Disease understanding” resulted in a loss of 40–45 million disability-adjusted life years per year for women, equivalent to a loss of four days of “healthy life” per year per woman worldwide.

In the context of endometriosis, lack of medical understanding hinders diagnosis as well as treatment. A study by academics at Manchester Metropolitan University, published in the Journal of Health Communication in January, interviewed British women at different stages of receiving a diagnosis, which takes an average of ten years. Many respondents said that their symptoms were initially (and sometimes repeatedly) dismissed as either normal menstrual pain, the result of lifestyle factors such as being overweight, or psychological. One reason why diagnosing endometriosis can be such a long, difficult process is that it almost always requires surgery: Most lesions can be found simply by inserting a camera (although those that cause cysts are usually detected on scan). appear to). To speed things up, scientists are looking for “biomarkers” – signatures of proteins or processes related to a disease that show up in an easy-to-test place, such as a patient’s blood or urine.

French pharmaceutical firm Zweig claims to have found such a solution for endometriosis. Its test looks for specific microRNAs – tiny strands of genetic material – which, one study shows, appear in the saliva of women with an existing endometriosis diagnosis. France’s health care authority in January approved a pilot plan to assess the effectiveness of Zweig’s “EndoTest” ahead of a potential rollout. In 2022, the country’s President Emmanuel Macron declared endometriosis a “problem of society” and made improving treatment a national priority. ,

These developments don’t mean the problem of diagnosis has been solved, cautions Dr. Horn, who believes more research is needed to test how endometriosis biomarkers show up in larger, more disparate populations. Is. But, after years of relative inactivity, he now sees endometriosis research as a “fast-moving field.” “I think I feel confident,” he says.

© 2024, The Economist Newspaper Limited. All rights reserved. From The Economist, published under license. Original content can be found at www.economist.com

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Published: April 12, 2024, 06:00 PM IST