Ozempic and other drugs like it have been shown to be effective in controlling blood sugar and promoting weight loss. Now researchers are investigating whether they could be just as transformative in treating a range of other conditions, from addiction and liver disease to general infertility.
It’s like a snowball that turned into an avalanche, said Lindsay Allen, a health economist at Northwestern Medicine. When drugs gain momentum, he said, they leave behind this completely reshaped landscape.
Many of the other uses of semaglutide, a compound in Ozempic and Wegovy, and tircepatid, a compound in Mounjaro and Zepbound, are only in the early stages. One of the biggest questions researchers are trying to answer: Are the benefits of these drugs just due to weight loss? Or do they have other effects, such as curbing inflammation in the body or silencing the brain’s obsessive thoughts, that would make it possible to treat many more diseases?
We’re unlikely to find out anytime soon. We were still learning how these drugs work, said Dr. Daniel Drucker, one of the first researchers to study these drugs. (Tr. Drucker consults for Novo Nordisk, Ozempic and the company that makes Wegovy.)
People with the conditions listed below, many of whom have few good treatment options, may benefit in the long term if these trials are successful. And for weight-loss drug manufacturers, each new use could catapult the drugs further into blockbuster status. Some of these applications, including heart disease and sleep apnea, which each affect tens of millions of people, have become targets for these companies and could prove particularly lucrative. These drugs are a goldmine, Dr. Allen said. There is no upper limit to where the market is going.
As evidence emerges from these studies, researchers will gain a clearer understanding of exactly how these drugs work in the body. If they can treat even more diseases, they could shake up medicine again.
Alcohol use disorder
Alcohol use disorder, also known as alcoholism, is common, with nearly 30 million people in the U.S. having the condition in 2022. But it’s rare for people to get a diagnosis, and it’s often extraordinarily difficult for them to find treatment. There are effective medications on the market, but some people who could benefit from them don’t even know they exist.
Anecdotally, some people who take drugs like Ozempic say that the drugs make them want to drink less and, in some cases, cut out alcohol altogether. Researchers are trying to figure out why. Because people feel full when taking these drugs, they may lose interest in alcohol and food. It is also possible that because these drugs target parts of the brain that control appetite, they may also affect compulsive behaviors that may involve those brain areas, such as alcohol or stimulant use, gambling, smoking, or even nail biting.
One small study followed six people with alcohol use disorder who used semaglutide for weight loss. All six drank significantly less when they had been using drugs for one to nine months. In an online survey of 153 obese adults, most of whom were Caucasian and female, those taking semaglutide or tircepatide reported drinking significantly less than others not taking the drugs.
Although data is limited, some patients with alcohol use disorders have already started asking doctors about these drugs. Researchers studying the effect of these drugs on drinking recently responded to this demand with a paper in Nature Medicine urging doctors not to use drugs for alcohol use disorders without further research.
Polycystic ovary syndrome
As many as five million people in the United States have polycystic ovary syndrome, or PCOS. The condition is a leading cause of infertility and causes irregular periods. Treatments with diet and exercise changes, birth control pills, and the diabetes drug metformin are available, but they don’t work for everyone.
Researchers believe that high testosterone levels contribute to PCOS. When people with the condition lose weight, their testosterone levels often drop. Drugs like Ozempic can help regulate hormones in PCOS patients, said Dr. Melanie Cree, who is leading one of the earliest studies to determine whether semaglutide can resolve PCOS symptoms.
A small study of 27 obese women with PCOS who took low-dose semaglutide found that after six months, most participants had lost weight and had more regular periods, suggesting that their PCOS was better under control. Dr. Cree completed a study of semaglutide in adolescent girls with PCOS that showed similar results, and is recruiting for another focused on menstrual regularity.
Up to 70 percent of people with type 2 diabetes and 50 to 90 percent of obese people have non-alcoholic fatty liver disease, or NAFLD, which occurs when excess fat builds up in the liver. The condition can cause liver damage so severe that some patients require liver transplants.
Doctors typically tell obese or overweight NAFLD patients to lose weight to reduce the amount of fat and inflammation in the liver. Because drugs like Ozempic make people lose weight, they can also reduce the amount of fat stored in the liver. Type 2 diabetes also increases the risk of NAFLD. Medicines like Ozempic can also reduce the risk or severity of liver disease by treating it.
Researchers have two other theories about how the drugs may help: by improving overall insulin resistance in NAFLD patients and by reducing inflammation that can damage the liver.
A study funded by Novo Nordisk showed that compared to a placebo, semaglutide did not significantly improve liver scarring or non-alcoholic steatohepatitis, or NASH, a severe non-alcoholic fatty liver disease. People with cirrhosis or a scarred and permanently damaged liver participated in the study. A larger study funded by Novo Nordisk found that patients who had previously received semaglutide were more likely to have improvement in NASH than patients who received placebo, but not significantly more likely to have improvement in scarring.
Novo Nordisk is now conducting an even larger clinical trial of semaglutide and NASH, and the Food and Drug Administration has designated the drug as a breakthrough treatment for the disease, speeding up regulatory review. Researchers funded by Eli Lilly are also investigating whether tircepatide can treat NASH.
Heart disease will kill nearly 700,000 people in the United States in 2022, making it the nation’s leading cause of death. Experts say there is an urgent need for new treatments that can reduce the risk of heart attacks and strokes and improve symptoms such as fatigue and shortness of breath that can make it harder for people with heart disease to get through the day.
Obesity significantly increases the risk of cardiovascular disease, which is why some doctors think that dieting can treat and prevent heart problems. Medicines can also help reduce inflammation, which can lead to plaque build-up in the heart and trigger blood clots.
In November, a large study showed that semaglutide reduced the risk of events such as heart attacks and strokes by 20 percent in obese and overweight people with heart disease. Another trial in obese people with certain types of heart failure found that semaglutide can improve symptoms and make exercise easier. Eli Lilly is conducting its own research on tircepatid and heart failure. And more studies are in the pipeline: Researchers are investigating whether semaglutide can reduce plaque in the heart, improve symptoms of heart failure, or reduce damage caused by strokes.
An estimated 30 million people in the United States suffer from obstructive sleep apnea, in which breathing stops and starts repeatedly during sleep. Treatments such as continuous positive airway pressure or the use of CPAP machines can help, although patients often find them uncomfortable. Many cases of sleep apnea go undiagnosed. If not treated properly, the condition can have a significant impact on people’s health and can increase the risk of complications such as heart disease and type 2 diabetes.
Sleep apnea is more common in obese people. This is due to a complex of factors, including the fact that fat deposits in the neck can block the airway when a person is lying down. Studies have shown that weight loss, including bariatric surgery, can help.
There is very little information so far, although an Eli Lilly spokesperson said the company is expected to complete a study of tircepatide and sleep apnea in the spring of 2024. Novo Nordisk is not currently studying whether semaglutide can treat sleep apnea.
About one in three adults with type 2 diabetes also suffer from chronic kidney disease, which occurs when the kidneys are damaged and unable to function properly. Patients with the condition may ultimately need dialysis or a kidney transplant, and if left untreated, the condition can be fatal.
Kidney damage occurs slowly over time and is irreversible in almost all cases, said Dr. George Bakris, who participated in the Novo Nordisk-supported semaglutide study in people with type 2 diabetes and chronic kidney disease. Drugs like Ozempic can prevent further damage in part because weight loss reduces the risk of hypertension and type 2 diabetes, which are risk factors for chronic kidney disease.
It’s not entirely clear how other drugs might improve chronic kidney disease, but one possible factor is that they can reduce inflammation, which can be harmfully high in people with the disease.
Novo Nordisk said in October that it had stopped a trial of semaglutide in people with type 2 diabetes and chronic kidney disease after an early analysis showed the drug was effective, although the company has yet to release the data. The company is funding another study looking at how semaglutide works in the kidneys. Eli Lilly is funding a trial of tircepatide in people with obesity and chronic kidney disease.
These and other studies are part of what Dr. Howard Forman, a Yale School of Medicine professor who specializes in health policy, calls an explosion of opportunity.
It remains to be seen whether these possibilities will turn into new forms of treatment.
We are very, very early in the rise of this whole industry, Dr. Forman said, and nothing surprises me.
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