
The “missed disease” that affects 190 million people: an endometriosis drug seems promising

Endometriosis, a disease in which uterine cells form lesions outside the uterus, has been called a “missed disease” because of its unknown cause, delays in diagnosis and disparities in treatment.Credit: Zephyr/Science Photo Library
A new treatment for endometriosis – a chronic, painful condition that around 190 million people live with – could be on the horizon.
The disease is caused by cells from the inner lining of the uterus growing in parts of the body where they do not belong. The resulting lesions are usually treated with surgery, hormone therapy, or a combination of the two. However, these options come with various complications and are often ineffective in the long term.

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Researchers have now modified niclosamide, a drug used to treat parasitic infections such as tapeworms, to target the cells involved in the disease, and tested it on mice. The study is published in Advanced healthcare materials1.
If replicated in humans, the findings could represent a “breakthrough” in treating endometriosis, says co-author Kanako Hayashi, a reproductive biologist at Washington State University in Pullman.
The study is a “nice first step” toward nonsurgical, nonhormonal therapies, says Elise Courtois, a molecular biologist at the Jackson lab in Farmington, Connecticut.
When treatment misses the target
Endometriosis can affect anyone born with a uterus and is most commonly diagnosed in girls and women between puberty and menopause.
People with this condition often experience severe abdominal and pelvic pain, which usually worsens during menstruation, as well as fertility problems. This disease is underdiagnosed and undertreated, in part because it can only be definitively diagnosed through surgery to identify lesions outside the uterus. Other factors include the normalization of period pain and the historic gender gap in health care.
Current treatments are far from adequate. Nearly half of people who undergo surgery to remove lesions – the gold standard treatment – experience a recurrence within five years. And hormonal therapies are disruptive; they often prevent pregnancy and side effects are wide ranging and include depression, hot flashes and osteoporosis.
Researchers are therefore looking for alternatives. In 2016, Hayashi worked on a study that found that the anti-inflammatory effects of niclosamide helped blunt the growth of implanted endometrial tissue in mice.2.
But there was a problem. Niclosamide is not well absorbed by the body due to its low water solubility. This means people have to take high doses for it to work, which often causes nausea, loss of appetite, and stomach cramps. In fact, the United States Food and Drug Administration has approved the use of niclosamide for only seven consecutive days. This is not practical for treating a chronic illness.
Gn Health