Image of an ovary showing polycystic ovary syndrome or PCOS
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Research led by Fudan University in Shanghai shows that artemisinin, which is used in anti-malarial drugs, shows promise for treating the symptoms of polycystic ovary syndrome.

Polycystic ovary syndrome is estimated to affect up to 13% of women of reproductive age but is known to be significantly underdiagnosed so rates may be higher in reality. It causes hormonal imbalance, with higher-than-normal levels of testosterone, irregular periods, and ovarian cysts, which can make becoming pregnant difficult, as well as weight gain and metabolic and skin related symptoms. Very few treatment options are currently available to people with this condition.

Chinese scientist Youyou Tu won the Nobel Prize in Physiology or Medicine jointly with William Campbell and Satoshi Ōmura in 2015 for the discovery of artemisinin and dihydroartemisinin and their use for treating malaria.

Qi-Qun Tang, a professor at Fudan University, and colleagues previously showed that artemisinin has the potential to increase energy expenditure and insulin sensitivity in addition to its antimalarial properties.

In the current study, published in Science, Tang and team also showed that artemisinin can reduce the level of androgen hormones such as testosterone, which are believed to be responsible for many symptoms of polycystic ovary syndrome.

The researchers studied both a mouse and a rat model of polycystic ovary syndrome and also carried out a small study in 19 women with the condition. They found that artemisinin blocks overproduction of testosterone by promoting CYP11A1 protein breakdown via an enzyme called LON peptidase 1 (LONP1). CYP11A1 helps to synthesize testosterone.

The study results showed that artemisinin targets LONP1, triggers overexpression of the gene and encourages interaction with CYP11A1, thus promoting its breakdown and lowering testosterone levels.

In the rodent models, treatment with artemisinin improved hormone levels, stopped irregular estrous cycles, reduced ovarian cysts and raised fertility levels. These effects were also seen in the trial in human patients. Testosterone and anti-Müllerian hormone levels were reduced, as well as the extent of ovarian cysts, and menstruation cycles were normalized.

“Overall, our findings highlight the promising potential of artemisinins as effective drugs for the comprehensive treatment of polycystic ovary syndrome… Our previous findings showed that artemisinins promoted metabolic homeostasis and protected against obesity, which prompted us to investigate whether artemisinins could regulate polycystic ovary syndrome development,” write the authors.

“In this study, we observed that artemisinins exhibited benefits to the reproductive endocrine in the polycystic ovary syndrome-like rodent models and patients, with efficacy against hyperandrogenism, irregular estrous cycles, and polycystic ovaries. However, in the preventive experiments, we did not observe an obvious metabolic effect of artemisinins in polycystic ovary syndrome-like mouse model, indicating that the effect of artemisinins… might not rely on the improvement of systemic metabolic state.”

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