California [US], September 16 (ANI): Researchers at the University of California San Diego School of Medicine have identified an investigational drug that shows promise in treating metabolic dysfunction-associated steatohepatitis (MASH), a severe form of fatty liver disease linked to obesity and type 2 diabetes that can lead to cirrhosis, liver failure, and even liver cancer.
The study, published August 23, 2025, in the online edition of The Lancet, found that the medication, ION224, targets a liver enzyme called DGAT2, which plays a key role in how the liver produces and stores fat. By blocking this enzyme, the drug helps reduce fat buildup and inflammation — two major drivers of liver damage in MASH.
“This study marks a pivotal advance in the fight against MASH,” said Rohit Loomba, MD, principal investigator of the study and chief of the Division of Gastroenterology and Hepatology at UC San Diego School of Medicine. “By blocking DGAT2, we’re interrupting the disease process at its root cause, stopping fat accumulation and inflammation right in the liver,” he added.
The multicenter, Phase IIb clinical trial involved 160 adults with MASH and early to moderate fibrosis across the United States. Participants received monthly injections of the drug at different doses or a placebo for one year. At the highest dose, 60 percent showed notable improvements in liver health compared to the placebo group. These benefits occurred regardless of weight change, suggesting the drug could be used alongside other therapies.
The medicine showed no serious side effects linked to the treatment.
MASH, formerly known as nonalcoholic steatohepatitis (NASH), affects people with metabolic conditions such as obesity and type 2 diabetes. It is often called a “silent” disease because it can progress for years without symptoms. More than 100 million people in the United States have some form of fatty liver disease, and as many as one in four adults worldwide may be affected, according to the Centers for Disease Control and Prevention. If left untreated, MASH can progress to liver failure and may require a transplant.
“This is the first drug of its kind to show real biological impact in MASH,” Loomba said. “If these findings are confirmed in Phase III trials, we may finally be able to offer patients a targeted therapy that halts and potentially reverses liver damage before it progresses to life-threatening stages,” he added.
Loomba, who is also director of the Metabolic-Dysfunction-Associated Steatotic Liver Disease (MASLD) Research Center at UC San Diego School of Medicine and a gastroenterologist and hepatologist at UC San Diego Health, said these results bring new hope for patients and families affected by the condition. He emphasized that early intervention and targeted therapies may also reduce the burden on health care systems by preventing costly and complex liver disease down the line. (ANI)
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