It took Allison Tuchman 12 years to get a correct diagnosis of polycystic ovary syndrome and another 10 years to find a treatment that would control the most severe symptoms. The 44-year-old Manalapan, New Jersey resident said she had finally found a drug that worked, and she said there was no turning back. “I will accept it until the end of time,” she said.
That drug happens to be semaglutide, the same drug that is in high demand mainly because of its effects. Effect on weight loss. It is approved for type 2 diabetes under the name Ozempic and for weight loss under the name Wegovy. Semaglutide is subdivided into: A class of drugs called GLP-1 agonistswhich includes diabetes drugs such as Mounjaro and Victoza.
Since the drug flooded the scene, other potential uses for them have been reported, in treating conditions ranging from polycystic ovarian syndrome to Alzheimer’s, Parkinson’s, and addiction. alcohol use disorderliver disease, and possibly cancer.
However, to prove that they work for individual symptoms, years of in-depth clinical research and large-scale clinical trials must be spent and enormous amounts of resources must be expended. We also need access to a reliable supply of these medicines, but this is by no means taken for granted. widespread shortages.
“Unfortunately, due to supply shortages, Novo Nordisk is focused on manufacturing medicines for clinical care, not for research,” said Lorezo Reggio, Ph.D., Clinical Director, National Institute on Drug Abuse, National Institutes of Health. said. He has studied these drugs as potential treatments for addiction and alcohol use disorders.
These constraints mean that the evidence needed to get the Food and Drug Administration, insurance companies, and even drug companies to participate in these new uses is for individuals like Tuchman, who says he’s seen first-hand that “drugs work.” It means that it lags behind the typical experience. .
“While these anecdotes from people reporting beneficial effects of semaglutide are very welcome, they are not the end of the story,” Reggio said. “Drug development requires rigorous science with extreme approaches such as clinical trials.”
Is Ozempic effective for PCOS?
Although a GLP-1 agonist has not been approved by the FDA to treat polycystic ovary syndrome, Tuchman has been on Ozempic off-label for three years. Tuchman said the drug has helped treat her more serious PCOS complications, including excess insulin production in the body, high blood pressure, and high blood pressure. cholesterol. Since taking her Ozempic, her blood sugar levels have stabilized and her debilitating fatigue, which used to be a strenuous task to lift her head off her pillow, has lessened.
Ann An estimated 5 million women in the US have PCOSis a poorly understood and often misdiagnosed condition that can lead to hypersecretion in women. testosterone. This can mean pain, heavy periods, irregularities, infertility, excess facial or body hair, severe acne, small ovarian cysts, and more. This hormonal imbalance can also lead to metabolic complications.
According to Dr. Melanie Cree, director of the multidisciplinary PCOS clinic at the University of Colorado, women with PCOS often develop insulin resistance, which can progress to diabetes if left untreated.Insulin resistance can also cause weight gain Even with diet and exercise, it becomes difficult to lose weight.
Doctors often prescribe metformin, another diabetes drug, to help with PCOS-related insulin resistance, but this drug works differently than GLP-1 agonists. Tuchman tried metformin for several years, but said it stopped working, so her doctor recommended Ozempic.
Cree is conducting a Phase 2/3 clinical trial of semaglutide in young women with both PCOS and obesity. Pediatric endocrinologists track participants’ weight loss, hormone levels, and the regularity of their menstrual cycles to determine whether the drugs are really helping their symptoms.
But Cree isn’t sure if semaglutide’s effect on PCOS is separate from its effect on weight, because losing weight in general may help treat PCOS. To help answer this question, she plans to take a closer look at the hormonal and metabolic consequences of the women in the study who took semaglutide but didn’t lose much weight. If the drug ameliorated the hormonal and metabolic effects of her PCOS but didn’t cause weight loss, the drug would have had an independent benefit for her PCOS, Cree said. Stated.
“When it works, it works”
Cree is also planning another large PCOS trial to further investigate whether semaglutide increases ovulation in women with PCOS.
The results of her current research are expected in the fall. Meanwhile, Dr. Cree, who also prescribes semaglutide to patients with diabetes and obesity, many of whom also have polycystic ovary syndrome, said her personal experience she had heard revealed. .
“When it works, it works,” she said. Cree remembers one of his patients’ mothers crying during an appointment late last year. “She said, ‘He gave me back her daughter,’ and she had everyone there crying,” Ms Cree said. “These drugs are life-changing.”
Rekha Kumar, Ph.D., an endocrinologist at Weill Cornell Medicine in New York City, believes that GLP-1 agonists like Ozempic could be a panacea for women with PCOS, even if they show clear benefits in clinical trials. warned that it would not. Symptoms can vary considerably and not every woman will experience the metabolic symptoms most likely to benefit from her GLP-1 agonist.
The drug is also much more expensive than existing drugs that address insulin resistance, such as metformin. “GLP-1 agonists can be used instead of metformin if the patient has never responded to metformin before or if the drug was not tolerated because of side effects,” Kumar said.
Trials like Cree’s may help compile evidence that these drugs work in PCOS, but FDA approval and Broad coverage insuranceThat would require the companies that make these drugs — Novo Nordisk in the case of semaglutide — to step in and conduct much larger and more expensive trials.
The company has no such plans, a spokesperson for Novo Nordisk confirmed in an email.
“To the best of my knowledge, there are pharmaceutical companies working to develop GLP-1 agonists for FDA approval for the PCOS indication,” said Dr. Andrea Dunaif, an endocrinologist at Mount Sinai Health System in New York. I am very sorry not to.” city. “GLP-1 agonists show great promise for the treatment of PCOS in overweight or obese women.”
Cree is pushing ahead with the PCOS trial anyway, but worries that widespread demand could complicate the planned August start date for the large trial.The shortage could affect Cree as well as patients because the University of Colorado needs to buy the drug from Novo’s Nordisk for Cree to use in trials. trying to fill a prescription. Ms Cree said Novo’s Nordisk told her that her drug shortage should be resolved by August.
“It’s particularly frustrating,” she said.
Meanwhile, she noticed a significant increase in women with PCOS asking about enrollment.
Explore other uses, including addiction
Besides PCOS, trials are underway to see if GLP-1 agonists can treat addiction and Alzheimer’s disease. Researchers are also investigating whether the drug works against Parkinson’s disease, sleep apnea, and an increasingly prevalent and serious condition called sleep apnea. nonalcoholic fatty liver disease. A group of Irish researchers have published a very small but interesting study in a journal. obesity Obese people who took Ozempic showed improved immune cells. These effects could theoretically mean a better chance of fighting off cancer, but experts say the studies are too early to say for sure.
In the addiction field, Joseph Schacht, an associate professor of psychiatry and drug addiction at the University of Colorado School of Medicine, is preparing to start a trial to see if semaglutide works. reduce cravings for alcohol More than placebo.
Schacht first became interested in conducting such a trial when a fellow psychiatrist found that some patients who had taken semaglutide for obesity had lost interest in alcohol altogether. It was when I mentioned
“It was very impressive to hear that patients had stopped drinking on their own accord,” he says. “I’ve been dealing with alcohol use disorder for a long time, and there aren’t many drugs that work for it.”
Schacht said a growing number of studies in rats and mice suggest this is the case, but studies in humans are still in the early stages.
When we delve deeper into the science behind these drugs, especially their effects on the brain, we learn how they work on problems ranging from alcohol and drugs to certain repetitive behaviors like nail biting. I learned.
In normal digestion, after a person has eaten, the small intestine releases the GLP-1 hormone, which causes the pancreas to release insulin into the blood, he explained. This insulin lowers blood sugar levels and sends a signal to the brain that the body is full and doesn’t need to eat anymore. GLP-1 agonist drugs work by mimicking that hormone and help lower blood sugar levels while making you feel full.
Interestingly, however, the feeling of ‘satisfied’ affects more than just the part of the brain that regulates appetite. craving for alcohol and drugs.
Professor Reggio of the NIH is also preparing to begin a human clinical trial of semaglutide for alcohol use disorders by the end of the year, and hopes to see positive results.
But without a trial, there is no evidence. And, as both Reggio and Schacht acknowledge, there are no trials without drugs.
Like Cree, who conducted trials for PCOS, both Leggio and Schacht are concerned that shortages will make it difficult to secure drugs for research. Nordisk said in an email that it has no immediate plans to study semaglutide for alcohol use disorders.
Reggio said even if supply issues were resolved, ultimately it would still be important to get the reins of research so drug companies would have the funding and resources to conduct large-scale clinical trials. added.
Pharmaceutical companies intervene in Alzheimer’s disease
There is one disease that intrigues Novo Nordisk enough to warrant a large drug trial. It’s Alzheimer’s disease.
Novo Nordisk is conducting two clinical trials involving approximately 4,000 people to see if semaglutide is superior to placebo. slows cognitive decline It is common in people with early Alzheimer’s disease.
Leila Parand, Ph.D., a neurologist who treats Alzheimer’s patients at UCLA Health, said previous research studies have shown that these drugs may help prevent damage to the brain vessels that can lead to Alzheimer’s disease. It is suggested that
“They preserve nerve cells, extend the growth of nerve cell branches, inflammation‘ said Parand. If effective, GLP-1 drugs would be a welcome addition to the limited treatment options for Alzheimer’s disease.
UCLA’s Paland Medical Center is one of hundreds of sites where patients can be enrolled in one of the Novo Nordisk clinical trials of semaglutide for early Alzheimer’s disease. Earlier this month, she had to close her registration early after the trial reached capacity ahead of schedule. Parland said this was also due to her surge in interest in these drugs and the fact that many people with early-stage Alzheimer’s disease became aware of the trial.
However, it is still years away from any definitive conclusions about all these potential uses of semaglutide. One of Novohi’s Nordisk Alzheimer’s trials, which began in 2021, is not expected to end until 2026.
Other questions about diabetes and obesity that researchers are still trying to answer are up in the air. Do I need to take GLP-1 drugs forever? what happens when they stopWhat are the long term side effects?
“We really don’t know. We don’t have the data, so there’s a lot to learn,” Cree said.follow NBC Health upon twitter & Facebook.
This article was originally published NBC News.com
