A medicine that has been used for decades to treat asthma may one day help people with a serious liver disease that affects millions around the world.
Researchers at the Medical University of South Carolina, also known as MUSC, have found early evidence that formoterol, a common asthma medication, could help reverse damage linked to MASH, a severe form of fatty liver disease.
The study was published in the journal npj Metabolic Health and Disease and has attracted attention because the drug involved is already widely used and has a long safety history in humans.
MASH stands for metabolic dysfunction-associated steatohepatitis. It is an advanced form of fatty liver disease in which fat builds up in the liver and causes ongoing inflammation and damage.
Over time, this damage can lead to scarring of the liver, known as fibrosis, and eventually cirrhosis, liver failure, or the need for a liver transplant.
Doctors are increasingly worried about MASH because it is becoming more common worldwide. Rising rates of obesity, type 2 diabetes, and metabolic problems are driving the increase. Many people do not know they have fatty liver disease until it becomes severe because the condition often causes few symptoms in the early stages.
At present, treatment options for MASH are still limited. Lifestyle changes such as weight loss, exercise, and healthier eating remain the main recommendations for many patients. Only a small number of medications have been approved to help treat the disease, and these drugs do not work equally well for everyone.
The MUSC discovery happened by accident during kidney disease research. Scientists were originally studying whether formoterol could help protect the kidneys from damage linked to diabetes.
During those experiments, they noticed something unexpected. The mice receiving the drug not only showed improvement in kidney injury but also appeared to have healthier livers with less fat buildup.
Dr. Joshua Lipschutz, director of the Division of Nephrology at MUSC and one of the study authors, said the team was surprised by the finding. The unexpected liver improvement encouraged the researchers to begin a second study focused specifically on MASH and fatty liver disease.
To investigate further, the researchers used mice fed a high-fat diet designed to mimic MASH in humans. They found that treatment with formoterol appeared to reverse fatty liver disease on several levels.
The researchers also studied what might be happening inside the cells. They found signs that the drug may improve the way cells produce and use energy.
According to the scientists, formoterol seemed to increase mitochondrial biogenesis. Mitochondria are often called the “powerhouses” of cells because they produce energy needed for the body to function.
When mitochondria do not work properly, cells can become damaged more easily, especially in diseases linked to metabolism such as diabetes and fatty liver disease. The researchers believe formoterol may help the mitochondria work better, which could protect organs from damage.
The team also looked at medical records from patients who were already taking beta-2 agonist drugs like formoterol for breathing conditions such as asthma or chronic obstructive pulmonary disease.
They found that these patients appeared to have lower rates of serious liver-related problems, including cirrhosis and death from all causes.
Although these findings are encouraging, the researchers stressed that the human data only show an association and do not prove that the drug directly caused the benefits.
One reason scientists are excited is because formoterol is already an approved medication with a long history of use. Developing entirely new drugs often takes many years and costs enormous amounts of money. Repurposing an existing drug could make treatment available much faster if future studies confirm the benefits.
Researchers are now conducting a clinical trial involving patients with diabetic kidney disease. Interestingly, many people with diabetic kidney disease also have MASH, since both conditions are closely linked to metabolic dysfunction and diabetes.
The researchers described the trial as a “two-for-one” opportunity because it may provide information about both kidney disease and liver disease at the same time.
Still, many important questions remain unanswered. The current findings are based mainly on animal studies, and drugs that work well in mice do not always succeed in humans.
Scientists still need to determine the best dose, how long treatment should continue, and whether inhaled formoterol can deliver enough drug to affect the liver in people.
The researchers also caution that no medication is completely free from risk. Even drugs with strong safety records can have side effects or unexpected problems when used for different conditions.
Even so, experts believe the findings are promising because MASH is becoming one of the most urgent liver health problems worldwide. Many patients eventually develop severe liver damage that can require transplantation.
This study offers hope that a familiar asthma drug may eventually become part of a safer and more affordable treatment approach for metabolic diseases affecting both the liver and kidneys.
The study was published in npj Metabolic Health and Disease.
The findings are exciting because they suggest that doctors may someday be able to reuse existing medications to treat complex metabolic diseases instead of developing entirely new drugs from scratch. However, patients should remember that this research is still in an early stage.
More human clinical trials are needed before formoterol can be recommended as a treatment for MASH. If future studies confirm the results, this could become an important step toward cheaper, faster, and more effective treatment options for millions of people with fatty liver disease.
If you care about liver health, please read studies about a diet that can treat fatty liver disease and obesity, and coffee drinkers may halve their risk of liver cancer.
For more information about liver health, please see recent studies that anti-inflammatory diet could help prevent fatty liver disease, and results showing vitamin D could help prevent non-alcoholic fatty liver disease.
Source: Medical University of South Carolina.
