Antimalarial drug may help treat polycystic ovarian syndrome, research suggests

An antimalarial drug used in ancient Chinese medicine could be an effective treatment for polycystic ovarian syndrome (PCOS), a groundbreaking study suggests.

The herbal extract artemisinin seemed to prevent the ovaries from producing too much testosterone, and women who took the drug for 12 weeks had more regular periods. The findings of the small trial by a Chinese team are being hailed as a potential breakthrough that could lead to a whole new approach to treating the condition that affects around one in 10 women.

“It’s very rare that you get a brand new development in a very important condition, so this is big,” said Dr Channa Jayasena, a clinical senior lecturer at Imperial College London, who was not involved in the study. “It’s a huge potential breakthrough and very welcome because women’s health issues have traditionally not received the attention that heart disease and cancer do.”

PCOS occurs when the ovaries produce too much testosterone. The hormonal imbalance disrupts ovulation, which can lead to irregular periods and affect fertility. There are other far-reaching health consequences, with many patients developing insulin resistance, which increases the risk of obesity, heart disease and diabetes. Higher testosterone levels can also cause excessive facial hair and acne.

“The name (of the syndrome) makes it sound like it only affects the ovaries, which is a disservice to women,” Jayasena said. “It is a disease that affects the entire body. You are more likely to have obesity, heart disease, too much hair, acne, as well as irregular periods and fertility problems.

Current treatments include the birth control pill, which suppresses testosterone production to help manage irregular periods and the cosmetic symptoms of PCOS. Fertility drugs or surgery can also help stimulate ovulation in women with PCOS who cannot become pregnant. However, these treatments are not completely effective and are not suitable for everyone.

The latest research, published in Sciencesuggests that artemisinin blocks an enzyme called CYP11A1 that is crucial for testosterone production in the ovaries. In a series of detailed experiments in mice and rats with a PCOS-like condition, scientists showed that the drug lowered testosterone and restored fertility. “This is an outstanding tour de force of work… the results are quite dramatic,” Jayasena said.

In patients, PCOS disrupts the ovulation cycle, where normally the follicles in the ovaries mature on an assembly line, with one follicle selected each month to fully mature and release an egg. In PCOS, the follicles are overactive, which means that many small follicles mature at the same time.

Professor Richard Anderson, head of obstetrics and gynecology at the University of Edinburgh, said: “They get stuck in a traffic jam and instead of one continuing to ovulate, they all remain in this state of partial development.” He said the results of the study looked “remarkably positive”.

In a pilot trial, 19 women with PCOS were given artemisinin for 12 weeks. They had a significant reduction in hormone levels, an ultrasound scan showed a reduction in follicle activity and menstruation was restored in 12 of the participants (63%), with no reported negative side effects.

Prof. Qi-qun Tang, who led the research at Fudan University in Shanghai, said: “Based on our findings, artemisinins are promising candidates for the treatment of PCOS because they strongly inhibit androgen synthesis in the ovaries, reduce immature follicles and (menstrual) ) improve cycle. .”

The women’s periods remained regular during an extended 12-week follow-up after they stopped taking the drug. “We are now extending this observation period to determine whether there is a relapse after an extended period without the drug,” Tang said, adding that the team was working to refine the dosage and timing of the medication in view of conducting a larger clinical study. process.

Anderson said: “It looks very exciting and what is intriguing is that it is a repurposing of a drug that has been used for a long time. It has a great track record as a medicine that people can take safely, so that’s a fantastic hurdle to overcome already.”

A larger study will still be needed to assess the potential risks of long-term use of the drug. There is also the question of whether the drug restores fertility. This can be a potential benefit, but there can also be serious risks associated with taking a hormone suppressing drug during pregnancy. “It could be a double-edged sword if it helps you get pregnant,” Jayasena said. “Then you have to show that it is safe during pregnancy.”