Inside the Clinical Trials Hunting for Better Fatty Liver Treatments

Federal researchers are actively testing new treatments for nonalcoholic fatty liver disease (NAFLD), now also called metabolic dysfunction-associated steatotic liver disease, through multiple clinical trials that could change how doctors treat this increasingly common condition. The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is leading efforts to find better ways to prevent, detect, and treat fatty liver disease, which affects millions of Americans.

What Have Recent Clinical Trials Discovered About Fatty Liver Treatment?

The NIDDK's nonalcoholic steatohepatitis (NASH) Clinical Research Network has completed several groundbreaking studies that reveal which treatments actually work. NASH is the more severe form of fatty liver disease that includes inflammation and can progress to cirrhosis. These trials have tested everything from vitamins to prescription medications, providing doctors with real evidence about what helps patients improve their liver function.

Three major studies have produced particularly important findings. The Pioglitazone or Vitamin E for NASH Study found that daily doses of natural vitamin E—the type found in food sources rather than synthetic versions—improved NASH in study participants overall. However, many participants taking pioglitazone gained weight, and not everyone improved with either treatment.

How Are Researchers Testing New Treatments for Fatty Liver Disease?

Current clinical trials are exploring multiple approaches to treating fatty liver disease. Researchers are studying how genes may increase the risk for NAFLD and examining how liver diseases develop and progress over time. The trials also focus on finding new treatments specifically for NASH, the more advanced form of fatty liver disease.

The research has produced several key findings across different patient groups:

• Adult Treatment Results: The PIVENS study showed that natural vitamin E improved NASH in adult participants, though pioglitazone also helped some features of the disease without definitive proof the improvements weren't random
• Pediatric Breakthrough: The Treatment of Nonalcoholic Fatty Liver Disease in Children trial found that natural vitamin E improved the most severe form of fatty liver disease in some children
• Prescription Drug Testing: The FLINT trial discovered that obeticholic acid treatment was associated with improved liver function in people with NASH, though it also increased itching and total cholesterol levels

What Do These Trial Results Mean for Patients?

The clinical trial results provide both hope and caution for people with fatty liver disease. Natural vitamin E has shown consistent benefits across both adult and pediatric studies, but researchers emphasize that more research is needed to determine whether long-term treatment with vitamin E causes any risks. The obeticholic acid findings have led to a large worldwide study that is still being evaluated to determine whether the drug is safe and effective for NASH treatment.

For patients interested in participating in current research, federally funded clinical studies for NAFLD are actively recruiting participants. These trials are part of ongoing medical research that helps doctors and researchers learn more about the disease and improve health care for future patients. However, anyone considering participation should always discuss clinical studies with their health care provider first.

The research continues to evolve as scientists work to understand the complex factors that contribute to fatty liver disease development and progression. These clinical trials represent the most rigorous way to test new treatments and ensure they are both safe and effective before becoming widely available to patients.