Groundbreaking Clinical Trials for Patients with Nonalcoholic Fatty Liver Disease

It causes fibrosis and eventually cirrhosis. It often remains undiagnosed until advanced stages of disease. And it represents up to 30% of liver transplants. But it has nothing to do with drinking.

Nonalcoholic fatty liver disease (NAFLD) is one of the biggest hidden health threats facing patients today. It develops in patients who never drink a drop of alcohol. Quite often, fatty liver does not show up on liver tests. In fact, patients can even develop cirrhosis from nonalcoholic steatohepatitis (NASH), an advanced form of NAFLD that includes inflammation and liver cell damage, while their liver tests remain normal.

Even if physicians can diagnose the disease, fatty liver remains a challenge to treat. Although patients benefit if they lose weight and get their diabetes under control, most find these options challenging. There is currently no FDA-approved medication for NAFLD.

Preliminary results of a phase III clinical trial with one of the treatments, obeticholic acid, for patients with NASH were encouraging because it decreases inflammation and fibrosis of the liver, but the FDA is awaiting additional data prior to granting approval. Andrew deLemos, M.D., and Robert Reindollar, M.D., both with the Center for Liver Disease and Transplant at Atrium Health, are participating in and enrolling patients in a variety of clinical trials with various treatments.

Phase II and III clinical trials

To help wage the war against fatty liver disease, the team of physicians and advanced practice providers (APPs) with the Center for Liver Disease and Transplant at Atrium Health is actively enrolling patients with NAFLD who have a certain level of fibrosis and inflammation in phase II and phase III clinical trials. Led by Mark Russo, M.P.H., M.D., deLemos and Reindollar, the trials are designed to identify medical treatment that will slow the progression of NASH and help prevent patients from developing cirrhosis and requiring a liver transplant.

The research team is conducting screening and testing to verify that patients meet clinical trial criteria. The team is currently enrolling five to 10 patients for each new study. To qualify, patients must have NAFLD with some level of inflammation and fibrosis in the liver. “Any patient you suspect may have fatty liver is a potential candidate for our trials,” said Reindollar. “We often use noninvasive tests, such as transient elastography, before pursuing a liver biopsy. A biopsy is usually a qualification requirement for the trials.”

A key benefit for patients is the attentive follow-up care provided. “We track the disease progression in trial participants over an extended period, sometimes for several years,” deLemos explained. “This is a major advantage for patients who want to be carefully monitored to ensure optimal care and treatment.” As the only transplant center in the Charlotte region, Atrium Health ensures access to a wide range of care.

The team is enrolling patients in clinical trials for less common liver diseases, including autoimmune liver diseases such as primary sclerosing cholangitis and primary biliary cholangitis. Enrollment is also open for natural history studies.

History of groundbreaking research

The Nobel Prize in Physiology or Medicine for 2020 was awarded to scientists from the U.S. and the U.K. who contributed to the identification of the hepatitis C virus. Chronic hepatitis C was formerly the leading cause of cirrhosis and liver cancer in the U.S. Over the years, Atrium Health investigators took an active role in groundbreaking research on antiviral therapy that has since proven to be highly effective in curing patients of hepatitis C.

“It’s very exciting to be part of the history and enrolling patients in the pivotal clinical-stage trials of medications with high cure rates,” Russo revealed. “In just two decades, we went from curing only 10% of hepatitis C patients with interferon therapy that had terrible side effects to curing up to 100% of patients with direct-acting antiviral drugs with few side effects. We hope to build on this work to find an effective treatment for fatty liver disease.”

Innovative tools and technology

The team of physicians and advanced practice providers with the Center for Liver Disease and Transplant at Atrium Health uses advanced tools to assess and monitor liver disease. For example, the team was the first in the region to start using vibration-controlled transient elastography, a noninvasive test that allows physicians to assess fibrosis without a biopsy. The team also uses magnetic resonance elastography during clinical trials to assess fibrosis.

To help primary care providers assess risk in patients with fatty liver disease, the team helped program a validated NAFLD risk assessment tool into the electronic medical record system. “Interfacing with the provider’s electronic health records, the tool performs simple calculations to see if patients could be at risk for cirrhosis,” related deLemos. “This tool can help physicians ensure that at-risk patients get timely care and treatment before their disease progresses.”

Patient referral contact

To refer a patient for a clinical trial, contact Gale Groseclose (Charlotte) by phone 704-355-4875 or email Gale.Groseclose@AtriumHealth.org or contact Heather Roberts (Huntersville) by phone 704-801-5202 or email Heather.Roberts@AtriumHealth.org.