The growing push to better understand and treat PCOS

Polycystic ovary syndrome affects millions of women in the US but remains poorly understood, leading to delayed diagnosis and limited treatment options. But there is hope yet.

In the US, an estimated 5–6 million women have PCOS. Yet it remains under-recognised and underdiagnosed. Photo: Alex Boyd

Allison Tuckman’s menstrual periods, which began at age 14, were accompanied by cramping, vomiting, and heavy bleeding—symptoms so severe they forced her to miss school. “Then came the cystic acne that was debilitating to my self-esteem and also extremely physically painful,” says Tuckman, who is now 47 and lives in New Jersey, on the east coast of the US. “I knew something was very wrong but I was unable to get any clear-cut answer from a doctor.”

When Tuckman was 16, her physician prescribed birth control pills to help alleviate her symptoms, but offered no explanation or information as to their cause. It was only when she was ready to start a family at 30 that a doctor suggested she might have polycystic ovary syndrome (PCOS).

She was referred to a reproductive endocrinologist, received fertility treatment, and went on to have a successful pregnancy. But after the birth of her daughter, Tuckman’s symptoms came raging back. She gained weight rapidly, her horrible periods returned, and she felt sluggish and tired all the time.

“I was also dealing with mental health issues. I could just tell something was wrong. It felt like my whole body was out of whack,” she says, explaining she could not carry another successful pregnancy.

Tuckman was initially led to believe that PCOS was solely a reproductive disorder, and none of her healthcare providers explained the long-term care she would need. After she began doing her own research she uncovered the full scope: PCOS is a complex hormonal condition that affects far more than fertility. It has lifelong impacts and increases the risk of a range of chronic conditions.

“Back then, [circa] 2010, there was very little online information and social media barely existed,” Tuckman says. “I took to message boards online, and it took several years before I found a doctor who could finally explain what PCOS was, along with all of its serious endocrine and metabolic consequences.”

PCOS an under-recognised condition

PCOS is the most common endocrine disorder affecting women of reproductive age—around 6–13% of women globally. In the US, an estimated 5–6 million women have the condition. Yet it remains under-recognised and underdiagnosed, leaving many of the millions of women lacking access to effective treatment options.

PCOS can be difficult to diagnose because many of its symptoms overlap and mimic those of other medical conditions. Plus, women's complaints and symptoms are frequently dismissed or downplayed in healthcare settings, and providers often receive minimal training on PCOS. The average woman may see three or four different clinicians before a correct diagnosis is made, and it can take two or more years from symptoms onset to diagnosis for many women.

In addition, studies also show that certain populations face higher risk of missed diagnosis in the US. These include Black women, those receiving Medicaid (public health insurance), non-English speakers, and women with a higher body mass index (BMI).

The hallmark features of PCOS are linked to elevated male hormone levels, or hyperandrogenism. The condition may manifest as excess hair growth on the face, chest, and abdomen; acne; or thinning or loss of scalp hair. Many women experience irregular menstrual cycles, often the result of infrequent or absent ovulation. PCOS is a leading cause of infertility.

While much of the focus centres on infertility and reproductive issues, the condition is associated with a number of serious long-term health risks.

“The condition is more common among those with a family history of PCOS, insulin resistance—a state in which the body’s cells respond poorly to insulin—and metabolic syndrome, which refers to a cluster of conditions including high blood pressure, elevated blood sugar, excess abdominal fat, and abnormal cholesterol levels,” explains Dr John Parker, an endocrinologist at Wilmington Health in North Carolina. He says PCOS is more frequently diagnosed in populations with higher rates of insulin resistance, including Hispanic, South Asian, and Black women.

Symptoms can develop at any age after puberty, but many women are diagnosed in their 20s and 30s. Like Tuckman, the catalyst is often an inability to conceive. “PCOS is a diagnosis of exclusion—other causes of elevated androgens, such as adrenal disorders, thyroid dysfunction, or rare tumours, must be ruled out before the diagnosis is confirmed,” Parker explains.

But while much of the focus centres on infertility and reproductive issues, the condition is associated with a number of serious long-term health risks. These include diabetes, high blood pressure, cardiovascular disease, endometrial cancer, sleep disorders such as sleep apnoea, and depression and anxiety.

Insulin resistance is a particular risk factor for diabetes, but higher androgen levels also up the risk. Excess insulin can cause weight gain, while fat in the abdominal area—known as visceral fat—can lead to high cholesterol. These factors increase the risk of cardiovascular disease in women with PCOS.

Obesity can be both a cause and a consequence of PCOS, explains Lynsey A. Johnson from the PCOS Sisters Telehealth Clinic & Wellness, which provides care for women with the condition. “This is because the excess androgens defining PCOS drive up insulin resistance, which increases fat deposition. This increases the stores of excess testosterone, further exacerbating an error in metabolism in a vicious cycle. It is very much a which-came-first situation, the chicken or the egg, as it is cyclic.”

Addressing complications of PCOS

PCOS is a chronic condition and cannot be cured. To date, there are no therapies that target its root causes. Instead, treatment focuses on addressing individual symptoms and complications that may arise. These include lifestyle interventions and drug therapies.

“The foundation of treatment for PCOS is lifestyle modification, including healthier eating, increased physical activity, and weight loss, particularly in women who are overweight or obese,” Parker says. “Even modest weight loss can help restore ovulation, regulate menstrual cycles, and improve insulin sensitivity.”

Dr Kathleen Scarbrough, a public health and preventive medicine specialist and an obstetrician-gynaecologist at Stony Brook Medicine, says lifestyle interventions also help to reduce the risk of associated conditions like diabetes, cardiovascular disease, and depression. “These include a balanced, plant-forward diet with lean proteins, regular physical activity, adequate sleep, and stress management,” she says.

But while lifestyle interventions are important, Scarbrough notes that medications can be prescribed, especially for insulin resistance or elevated androgens. “Ongoing support for weight management and preventive care is essential across the lifespan of women with PCOS,” she says.

The types of medications vary. The first-line treatment for menstrual irregularity and hirsutism is usually combined oral contraceptives. Other treatments may include anti-androgen agents, or weight loss medications such as glucagon-like peptide 1 (GLP-1) receptor agonists.

Drugs to treat PCOS are considered off-label, since they lack regulatory approval for this indication. This can result in considerable cost for patients.

Restoring regular ovulation as well as addressing obesity can lower the risk of developing serious complications. “While PCOS does not change how conditions like heart disease are treated, improving the underlying metabolic disturbances associated with PCOS can strengthen treatment responses and help slow disease progression,” Parker says, explaining that women with PCOS should also be routinely assessed for depression and anxiety.

The trouble is that in the US, drugs to treat PCOS are considered off-label, since they lack regulatory approval for this indication. This can result in considerable cost for patients as health insurance may not cover them as insurers have a degree of discretion. One example is semaglutide, a GLP-1 agonist that was initially developed for managing type 2 diabetes but has shown promise in helping women with PCOS lose weight.

“FDA approval would increase insurance coverage for medications included in the PCOS treatment guidelines,” Johnson explains.

The lack of FDA approval has affected Tuckman’s access to a GLP-1 medication she has been using for almost 10 years, as she can no longer afford it. “I have severe insulin resistance. As of this week my insurance company is refusing to pay for the medication because technically it is not approved unless you are a type 2 diabetic.”

Exploring targeted PCOS treatments

Ongoing research is exploring treatments that target the root causes of PCOS. “Investigational treatments include novel insulin-sensitising agents, which are drugs that help the body use insulin better, medications that reduce inflammation, and interventions that target the gut microbiome,” Scarborough says.

Early results looks promising. A pilot clinical study by Fudan University in China found that artemisinins, which are compounds used to treat malaria, target many of the aspects of PCOS. Testosterone and anti-Müllerian hormone levels dropped, ovarian cysts were reduced, and the majority of the women resumed normal menstrual cycles.

“I’m not sure it will become a treatment but it definitively [warrants] more research,” says Elisabet Stener-Victorin, professor in the Department of Physiology and Pharmacology at the Karolinska Institutet in Sweden.

To date, there are no therapies that target the root causes of PCOS. Photo: Sharon Waldron

Stener-Victorin and her colleagues are also conducting preclinical and clinical investigations of PCOS. Last year they published a paper suggesting that elevated androgen levels during pregnancy affect the placenta and foetal germ cells, which are the precursors to sperm and eggs, in mice. However, treatment with an androgen receptor antagonist, an agent that keeps androgens from binding to proteins called androgen receptors, restored normal function to the placenta and germ cells.

Still, developing new treatments for PCOS is a slow process. “The main reason is that we still don’t know the mechanisms [involved in PCOS],” explains Stener-Victorin.

Limited research funding a major barrier

The lack of specific PCOS treatments also likely stems, at least in part, from a lack of research funding. Despite the need for better and targeted treatments, research remains chronically underfunded. “This is an urgent concern, especially in light of recent National Institutes of Health (NIH) budget cuts,” says Scarbrough. “Despite affecting millions of women, PCOS remains underfunded and under-researched compared to other chronic conditions.”

Among more than 300 medical conditions that receive funding from the NIH—the primary research agency in the US—PCOS is close to the bottom, with only about US$10 million allocated for research in 2024. Breast and prostate cancer, in comparison, received US$792 million and US$305 million, respectively. Up until 2022, PCOS funding was so low that it was not even included in the NIH funding list.

Johnson points out that conditions that affect only women are historically underfunded. Women were not included in research studies until 1985—and their inclusion was not mandatory until 1993. This is compounded by other persistent inequities. “There are also disparities in PCOS diagnosis, treatment, and funding based upon socioeconomics, race, and ethnicity,” Johnson says.

Contributing factors include geographic and financial barriers, implicit bias, and limited access to specialists. “Addressing these gaps will require better provider education, more inclusive research, and improving access to care,” says Scarbrough.