Antimalarial drugs could also treat polycystic ovary syndrome

Polycystic ovary syndrome (PCOS) is an endocrine disorder that affects millions of people worldwide, including about 8–13% of reproductive-aged women, according to the World Health Organization. People with PCOS have higher than normal levels of progesterone, testosterone, and other androgens, which can lead to irregular menstrual periods, ovarian cysts, infertility, and a higher risk for metabolic disorders. Current treatments address only the symptoms of PCOS, not the underlying hormone imbalance.

Now a team led by Qi-Qun Tang of the Fudan University School of Basic Medical Sciences has reported in Science that derivatives of the malaria treatment artemisinin, may also be effective against PCOS (2024, DOI: 10.1126/science.adk5382).

The researchers found that injections of the artemisinin derivative artemether lowered ovarian testosterone levels in rodent models of PCOS, improved the appearance of the animals’ ovarian tissues, and regulated their estrous cycles. They determined through cell studies that the drug works by promoting the degradation of a key androgen-producing enzyme.

The researchers also did a pilot clinical study of 19 women with PCOS. Taking dihydroartemisinin pills for 12 weeks reduced most of the participants’ androgen levels, and more than half of the participants had more-regular menstrual cycles after receiving the treatment. Tang says he and his team are now optimizing doses and timing for a larger clinical trial.

Elisabet Stener-Victorin, who researches PCOS at the Karolinska Institute, writes in a perspective article that she would like to see more investigation of the metabolic and long-term effects of artemisinin-based PCOS treatment, but adds that this study “represents a promising new approach . . . to improve the quality of life of millions.”