Scientists make 'pivotal advance' in fight against fatty liver disease as new drug offers hope to millions

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Solen Le Net

By Solen Le Net


Published: 16/09/2025

- 12:08

Sixty per cent of patients saw significant improvements in their liver health at a higher dose during the trial

Scientists have found a new drug that could help millions of people with a dangerous liver condition. The medication, called ION224, has shown remarkable results in treating MASH - a severe fatty liver disease that's linked to obesity and diabetes.

It's a condition that often goes unnoticed for years but can lead to liver failure and cancer. More than 100 million Americans have some type of fatty liver disease, and about one in four adults globally might be affected.


The breakthrough comes from researchers at UC San Diego School of Medicine, who have just published their findings in The Lancet.

The drug works by targeting something called DGAT2, a liver enzyme that controls how the body makes and stores fat.

FATTY LIVER DISEASE

Fatty liver disease can go unnoticed for years

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When ION224 blocks this enzyme, it stops fat from building up in the liver and reduces inflammation - the two main culprits behind liver damage in MASH.

"By blocking DGAT2, we're interrupting the disease process at its root cause, stopping fat accumulation and inflammation right in the liver," said Dr Rohit Loomba, who led the study at UC San Diego.

It's the first medication of its type to show this kind of biological impact on MASH.

The year-long clinical trial tested ION224 on 160 adults with MASH and early to moderate liver scarring across America. Patients received monthly injections at various doses or a placebo.

The results were impressive - at the highest dose, 60 per cent of patients saw significant improvements in their liver health compared to those on placebo.

What's particularly exciting is that these benefits happened whether patients lost weight or not, meaning the drug could work alongside other treatments.

Even better news: the medication didn't cause any serious side effects during the trial. This safety profile makes it a promising option for future treatment.

MASH used to be known as NASH (nonalcoholic steatohepatitis), and it's often called a "silent" disease because people don't realise they have it until serious damage has occurred.

FATTY LIVER DISEASE ILLUSTRATION

The therapy could reverse liver damage before it progresses to life-threatening stages

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"This study marks a pivotal advance in the fight against MASH," Dr Loomba explained.

"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."

For patients and families dealing with this serious condition, these results bring real hope for better treatment options.

Early intervention with targeted therapies like ION224 could also help reduce pressure on healthcare systems by preventing expensive and complex liver disease complications.