A new investigational drug may finally bring hope to people living with a serious form of fatty liver disease that has few treatment options today.
Researchers at the University of California San Diego School of Medicine have reported encouraging results from a clinical trial of a medication called ION224, the first drug to directly target an enzyme driving the disease process.
The condition, now called metabolic dysfunction-associated steatohepatitis (MASH), was previously known as NASH.
It is closely linked to obesity and type 2 diabetes and is marked by fat building up in the liver, along with inflammation and scarring.
Over time, MASH can lead to cirrhosis, liver failure, or even liver cancer. Because it often develops silently without symptoms, many people don’t know they have it until the damage is advanced.
According to the Centers for Disease Control and Prevention, more than 100 million people in the United States have some form of fatty liver disease, and worldwide as many as one in four adults may be affected. Yet there are currently no approved treatments that directly reverse liver damage.
The new study, published in The Lancet, tested ION224, which works by blocking a liver enzyme called DGAT2.
This enzyme plays a central role in how the liver produces and stores fat. By shutting it down, the drug reduces fat accumulation and inflammation, two of the main drivers of liver injury in MASH.
“This study marks a pivotal advance in the fight against MASH,” said Dr. Rohit Loomba, lead investigator and chief of the Division of Gastroenterology and Hepatology at UC San Diego School of Medicine. “By blocking DGAT2, we’re interrupting the disease at its root cause.”
The Phase IIb clinical trial involved 160 adults across the United States who had MASH with early to moderate scarring. Participants received monthly injections of either ION224 at different doses or a placebo for one year.
At the highest dose, 60% of patients showed meaningful improvements in liver health compared to the placebo group. Importantly, the benefits occurred regardless of changes in weight, showing that the drug works independently of weight loss. The treatment was also well tolerated, with no serious side effects linked to the medication.
Loomba explained that this is the first drug to demonstrate real biological impact in MASH. If results are confirmed in larger Phase III trials, ION224 could become the first targeted therapy to halt or even reverse liver damage before it reaches life-threatening stages.
For patients and families affected by this “silent” disease, the findings bring new hope. Loomba stressed that early intervention with targeted therapies like ION224 could not only improve outcomes for individuals but also ease the burden on health systems by preventing costly complications such as liver failure and transplants.
The next step will be to launch larger studies to confirm the drug’s effectiveness and move closer to making it widely available.
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Source: UC San Diego.
