Endometriosis-like symptoms can be treated in mice by targeting a gene that has been linked to the condition.
Endometriosis occurs when tissue from the uterus spreads to other areas like the ovaries and bladder. It affects up to one in 10 women and can cause intense pelvic pain and infertility.
The available treatments 鈥� which include surgery and injectable hormones 鈥� often don鈥檛 work and can cause unpleasant side effects.
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To better understand the condition, at Monash University in Melbourne, Australia, and his colleagues studied 100 women with endometriosis who each had several relatives with the condition, hinting at a genetic influence.
About a quarter of the women had variations in a gene called NPSR1. Analysis of another 3000 women with endometriosis showed that those with moderate to severe disease were also more likely to have variations in the NPSR1 gene.
Other NPSR1 gene variants have been linked with inflammatory conditions like asthma, allergies and arthritis, making it plausible that the gene plays a role in the chronic inflammation associated with endometriosis, says Tapmeier.
Following this discovery, the researchers wondered if targeting NPSR1 could help to treat endometriosis.
To explore this idea, they gave female mice endometriosis-like symptoms by implanting uterus tissue in other parts of their pelvises. They then treated them with a molecule called SHA 68R, which inhibits the receptor encoded by NPSR1.
The mice treated with the inhibitor appeared to have less abdominal pain than untreated mice and had lower levels of inflammation. This hints that targeting NPSR1聽may also help women with endometriosis, says Tapmeier.
However, the approach may not work for all patients, since not everyone with endometriosis has a variation in the NPSR1 gene, he says. It鈥檚 also unclear whether treatment would cause side effects, he says.
鈥淚t would be really nice if this holds up as an additional treatment option for women with endometriosis but it鈥檚 still early days,鈥� says Tapmeier.
The team now hopes to test the approach in female rhesus macaques, since endometriosis occurs naturally in some of these animals and seems to have a similar genetic basis as in people.
Science Translational Medicine
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