Scientists from the University of California San Diego School of Medicine have found a new experimental drug that could help treat a serious liver condition called MASH.
This disease, also known as metabolic dysfunction-associated steatohepatitis, is a severe type of fatty liver disease often linked to obesity and type 2 diabetes. If untreated, it can lead to liver scarring (cirrhosis), liver failure, or even liver cancer.
The study was published in the medical journal The Lancet. It shows that the drug, named ION224, works by blocking a liver enzyme called DGAT2. This enzyme helps the liver make and store fat.
When the drug blocks DGAT2, it reduces the amount of fat and inflammation in the liver. Both of these problems are known to cause liver damage in people with MASH.
Dr. Rohit Loomba, the lead researcher of the study, said this drug could be a major breakthrough in treating MASH. He explained that by stopping fat buildup and inflammation in the liver, the disease can be slowed down or even stopped early on.
The clinical trial involved 160 adults in the United States who had MASH and early or moderate liver scarring. Participants were given monthly injections of either the new drug or a placebo (a substance with no active drug) over a year. The results were promising.
At the highest dose of ION224, 60% of participants showed clear improvements in liver health compared to those who got the placebo. These benefits were seen even if the patients did not lose weight, meaning the drug could be effective alongside other treatments. Importantly, there were no serious side effects reported from the drug.
MASH, which used to be called NASH (non-alcoholic steatohepatitis), is often called a “silent” disease because it can develop slowly without noticeable symptoms. Many people don’t know they have it until serious damage has occurred.
In the U.S. alone, over 100 million people have some form of fatty liver disease. Globally, around one in four adults may be affected. Without treatment, MASH can lead to end-stage liver disease that may require a transplant.
Dr. Loomba, who also directs the MASLD Research Center at UC San Diego and works as a liver specialist at UC San Diego Health, said this is the first time a drug has shown a clear biological impact in treating MASH. If the results hold true in larger Phase III trials, this drug could become the first targeted treatment to help reverse liver damage in people with the disease.
For patients and families affected by MASH, this research offers new hope. Dr. Loomba added that early treatment and targeted drugs like ION224 may also ease the pressure on healthcare systems by preventing the need for expensive and complex care later on.
The next step for researchers is to begin larger trials to confirm these results and move closer to making the drug available to the public.
If you care about liver health, please read studies about simple habit that could give you a healthy liver, and common diabetes drug that may reverse liver inflammation.
For more information about health, please see recent studies about simple blood test that could detect your risk of fatty liver disease, and results showing this green diet may strongly lower non-alcoholic fatty liver disease.
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