A study published in Science on June 14 suggests that a common antimalarial drug called artemisinin could be used to treat polycystic ovarian syndrome (PCOS), an endocrine disorder that affects millions of people of reproductive age worldwide. Researchers found that after receiving repeated doses of artemisinin, a small group of women with PCOS had lower blood testosterone levels and healthier-looking ovaries, indicating that artemisinin may help alleviate symptoms of PCOS. Artemisinin compounds are already known to be effective against malaria, lupus, and cancer, and this discovery opens up the possibility of using them to treat PCOS as well.

The study has the potential to change the landscape of PCOS treatment, according to Elisabet Stener-Victorin of the Karolinska Institute in Stockholm. PCOS is a prevalent disorder characterized by elevated androgen levels and the presence of ovarian cysts. The exact cause of PCOS is unknown, and treatments typically target individual symptoms such as acne, excess hair growth, and infertility. People with PCOS are also at higher risk of metabolic conditions like type 2 diabetes, and researchers believe that underlying metabolic changes may contribute to the disorder.

The research team, led by molecular biologist Qi-Qun Tang from Fudan University in Shanghai, initially identified artemisinin as a potential treatment for obesity by converting fat cells into more metabolically active tissue. They later found that brown adipose tissue, which can be induced by artemisinin, could also be used to treat PCOS in rats. Animal studies showed promising results, prompting the team to conduct a small clinical trial with patients diagnosed with PCOS.

Nineteen PCOS patients received dihydroartemisinin, a form of artemisinin used in malaria treatment, three times a day for three months. Following the treatment, 12 patients experienced regular menstrual cycles, and most had reduced blood testosterone levels and fewer developing ovarian follicles. Artemisinin was found to inhibit the production of testosterone by degrading the CYP11A1 protein, which contains heme. This process results in lower testosterone levels and a reduction in ovarian cysts, a hallmark of PCOS.

While these initial findings are promising, further research is needed to validate the results and understand how artemisinin acts in the body. Sireesha Reddy, an obstetrician and gynecologist at Texas Tech University Health Sciences Center El Paso, recommends investigating how artemisinin affects cardiometabolic symptoms of PCOS and its impact on fertility. The research team plans to explore different forms and dosages of artemisinin in larger clinical trials to determine the best approach for treating PCOS and potentially restoring fertility in affected individuals.

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