The first endometriosis drug in four decades is on the horizon

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i amn 1690 Daniel Shrone, a German physician, described a patient with “ulcers” throughout her peritoneum, bladder, intestines, uterus, and cervix. This was believed to be the first documented appearance in the medical literature of endometriosis, a painful and debilitating gynecological condition that today affects 190 million women worldwide.

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. If endometrial tissue grows abnormally outside the uterus, it can cause destruction. In extreme cases of endometriosis, the glue can “bind” a woman’s organs together — from the ovaries to the bladder to the intestines — and cause them to clot in place. Mild cases can cause severe pain, heavy periods, inflammation and internal bleeding, fatigue and infertility due to scar tissue. There is no known cure, 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 in their lifetime – roughly the same as the proportion of the global population with diabetes. But while doctors understand what causes diabetes and how to treat it, their understanding of endometriosis lags “30 to 40 years,” according to Andrew Horne, professor of gynecology and reproductive sciences at the University of Edinburgh and president-elect of the World Endometriosis Society. He blames the lack of research and awareness, driven by a lack of funding.

Things started to change. 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, grew out of a closer examination of how endometriosis lesions form. By taking samples from patients during diagnostic laparoscopy, his team found that those with peritoneal endometriosis – meaning disease of the lining of the pelvic cavity, which represents about 80% of cases – had significantly higher levels of a chemical called lactate in their pelvis. Except.

Lactate is produced when glucose is broken down in the body (and is the cause of the uncomfortable stitches that can suddenly hit runners). Its increased presence, the researchers calculated, suggested a hand in the development of endometriosis lesions, perhaps similar to lactate’s role in helping cancer cells proliferate. The scientists then looked for a drug that had already been tested in cancer patients, eventually dichloroacetate (DCA) 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 were treated with DCA Less pain and better quality of life have been reported. A larger cohort, followed by an experiment with a placebo arm. If the drug is approved, which could be possible in the next five to seven years, DCA It will be the first new endometriosis treatment discovered in four decades.

“There’s still a problem — and I hate to say it — with problems that only affect women,” Dr. Horn said. That observation appears elsewhere. A report released last month by McKinsey, a consultancy, concluded that a “systematic lack of disease understanding” has resulted in an annual loss of 40m-45m disability-life-years for women, amounting to four days of “healthy life” lost. per woman every year worldwide.

In terms of endometriosis, lack of medical understanding hinders diagnosis as well as treatment. The study, conducted by academics at Manchester Metropolitan University, was published in January Journal of Health Communication, British women interviewed are at different stages of receiving a diagnosis, which takes an average of ten years. Many respondents said their symptoms were initially (and sometimes repeatedly) dismissed as either normal period pain, the result of lifestyle factors such as being overweight, or psychological. One reason endometriosis is diagnosed is such a drawn-out, difficult process that it almost always requires surgery: most lesions can only be found by inserting a camera (although the cause of the cyst is usually seen on scans). To speed things up, scientists are looking for “biomarkers” — proteins or signatures of disease-related processes that are as easy to test as in a patient’s blood or urine.

A French pharmaceutical firm called Zewig claims to have found such a solution to endometriosis. Its test looks for specific microRNAs—small strands of genetic material—that, one study found, appeared in the saliva of women diagnosed with existing endometriosis. In January, France’s health-care authorities approved a pilot scheme to assess the effectiveness of Ziwig’s “Endotest” before a possible rollout. In 2022, the country’s president, Emmanuel Macron, declared endometriosis a “societal problem” and made improving treatment a national priority.

This development does not mean that the problem of diagnosis has been solved, cautions Dr. Horn, who believes that more research is needed to examine how endometriosis biomarkers appear in larger, more disparate populations. But, after years of relative inactivity, he now sees endometriosis research as finally “a fast-moving field.” “I think I feel confident,” he says.

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