A malaria drug could be used to treat PCOS, a common hormone disorder

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Can a Malaria Drug Transform PCOS Treatment?

Polycystic ovary syndrome (PCOS) is one of the most common endocrine disorders, affecting up to 13% of women of reproductive age worldwide. Despite its prevalence, the condition remains poorly understood, with treatments that mostly manage symptoms rather than the root cause.

Now, a surprising discovery is making headlines: artemisinin, a drug derived from the Artemisia annua plant and widely used against malaria, may also hold the key to managing PCOS.

What is PCOS?

In polycystic ovarian syndrome, cysts form from follicles holding unreleased eggs (right ovary in this illustration compared with a healthy ovary, left). PCOS affects hundreds of millions of people worldwide, but effective treatments remain elusive.
Kateryna Kon/Science Photo Library/Getty Images

Why Artemisinin?

  • Origin: Extracted from the Artemisia annua plant.

  • Nobel Prize-winning drug: Revolutionized malaria treatment.

  • Also effective in: Lupus, certain cancers, metabolic disorders.

  • Discovery for PCOS: While screening 3,000 compounds, researchers at Fudan University (Shanghai) found artemisinin could transform white fat into brown fat (energy-burning tissue). Around the same time, scientists linked brown fat activity to improvements in PCOS in rats.
    Note:
    White fat: Stores energy, harder to burn, linked to obesity and insulin resistance.
    Brown fat: Burns energy to produce heat, improves metabolism, helps regulate blood sugar.
    Artemisinin effect: In lab studies, the drug activated pathways in fat cells that convert white adipose tissue into brown/beige adipose tissue.
    Why it matters for PCOS: Since many women with PCOS struggle with obesity and insulin resistance, this conversion could ease metabolic problems, improving both weight control and hormonal balance.
    In short: Artemisinin helps “reprogram” fat to behave in a healthier, energy-burning way, which may also reduce PCOS symptoms.

This overlap raised a critical question: could artemisinin also help women with PCOS?

How the Research Was Done

1. Animal Studies

  • Female mice were injected with excess androgens to mimic PCOS.

  • With artemisinin:

    • No ovarian cysts developed

    • Testosterone levels stayed lower

    • Fertility cycles remained normal

  • Similar outcomes were confirmed in rats.

2. Pilot Human Study

  • Participants: 19 women with PCOS.

  • Treatment: Dihydroartemisinin (the clinical form used in malaria) taken 3 times daily for 12 weeks.

  • Results:

    • 12 women regained regular menstrual cycles

    • Testosterone levels dropped in most patients

    • Ovaries showed fewer follicles, suggesting improved ovulatory health

How Does Artemisinin Work?

  • Key mechanism: Breaks down a testosterone-producing protein called CYP11A1.

  • Heme interaction: CYP11A1 contains heme (iron-based molecule). Artemisinin reacts with heme, producing reactive oxygen species.

  • Effect:

    • In malaria → kills parasites.

    • In cancer → damages tumor cells.

    • In PCOS → reduces testosterone, restoring ovarian balance.

This common mechanism explains why artemisinin works across such different diseases.

What Experts Are Saying

  • Dr. Elisabet Stener-Victorin (Karolinska Institute):
    “This could potentially change the landscape of PCOS treatment. Although more studies are needed, it represents a promising new approach.”

  • Dr. Gemma Casals (Hospital Clínic Barcelona):

    • Praised the study for not only reversing symptoms but also deepening knowledge of PCOS biology.

    • Stressed the need for longer studies in more diverse patient groups (e.g., women with obesity).

    • Compared findings to bariatric surgery outcomes, which also improve hormones in women with PCOS.

    • Highlighted ongoing research into vitamins, antioxidants, and probiotics as additional supportive treatments.

  • Dr. Sireesha Reddy (Texas Tech University):

    • Warned results are preliminary.

    • Called for studies on insulin, lipid levels, and fertility outcomes.

    • Recommended testing in women with higher BMIs and more severe PCOS.

Why This Discovery Matters

  1. Addresses the root cause: Unlike current drugs, artemisinin lowers androgens directly.

  2. Improves multiple symptoms: Menstrual irregularities, excess follicles, high testosterone.

  3. Repurposing advantage: Already approved for malaria, so its safety profile is well documented.

  4. Potential fertility restoration: Still under investigation, but results in mice suggest promise.

  5. Global relevance: PCOS affects millions; an affordable, accessible treatment could be life-changing.

The Road Ahead

Researchers are now focused on:

  • Testing different forms and dosages of artemisinin.

  • Running longer, larger clinical trials.

  • Examining effects in women with obesity and severe PCOS.

  • Studying its impact on fertility, insulin resistance, and cardiovascular risks.

Lead investigator Qi-Qun Tang emphasizes:

“We have a long way to go to see how they can get fertility back.”

Final Takeaway

  • PCOS is complex, widespread, and often misunderstood.

  • Artemisinin offers a glimmer of hope as a treatment that doesn’t just mask symptoms but targets hormonal imbalance at its root.

  • While it is not yet ready for clinical use in PCOS, the science is strong enough to warrant excitement — and caution.

For now, lifestyle management and existing medications remain essential, but the repurposing of this Nobel Prize–winning malaria drug could mark the beginning of a new era in PCOS treatment.

Bottom line: An old antimalarial drug might soon become a new weapon in women’s reproductive health, transforming how we understand and manage PCOS.

Citations:

Y. Liu et al. Artemisinins ameliorate polycystic ovarian syndrome by mediating LONP1-CYP11A1 interaction. Science. Vol. 384, June 14, 2024, p. 1187. doi: 10.1126/science.adh5382. 

E. Stener-Victorin. Remedy hope for polycystic ovary syndrome. Science. Vol. 384, June 14, 2024, p. 1174. doi: 10.1126/science.adq0328

J. Visser. The importance of metabolic dysfunction in polycystic ovary syndrome. Nature Reviews Endocrinology, Vol. 17, February 2021, p. 77-78. doi: 10.1038/s41574-020-00456-z.

P. Lu et al. Artemisinin derivatives prevent obesity by inducing browning of WAT and enhancing BAT function. Cell Research, published online September 16, 2016. doi: 10.1038/cr.2016.108

X. Yuan et al. Brown adipose tissue transplantation ameliorates polycystic ovary syndrome. Proceedings of the National Academy of Sciences, published online February 22, 2016. doi: 10.1073/pnas.1523236113.

https://www.clinicbarcelona.org/en/news/a-compound-against-malaria-possible-treatment-for-polycystic-ovary-syndrome

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