Liver diseases have recently emerged as a global health priority, and nonalcoholic fatty liver disease (NAFLD) has become the most common chronic liver disease in children worldwide. But medical treatments for NAFLD are not yet available to children and young adults. A recent paper produced by The Liver Forums’ Pediatric Working Group and published in the journal Gastroenterology combs through the research to look for the best treatment— a “silver bullet”—to treat pediatric nonalcoholic diseases drug development.
In the U.S., the CDC estimates that just over 20% of adolescents can be classified as obese; nearly 40 percent of these adolescents are estimated to have NAFLD. Without effective treatment, children with nonalcoholic steatohepatitis, a type of NAFLD, are at risk of developing cirrhosis and experiencing liver-related mortality in early adulthood.
“It is a tricky field because we need to be able to measure the impact of a new drug on a complicated condition,” said Veronica Miller, a coauthor of the study and an adjunct professor at UC Berkeley School of Public Health.
A big challenge in measuring the efficacy of pediatric NAFLD drugs is identifying the liver disease biomarker, or the physical indicator of the disease, such as elevated enzyme levels, in children. Without a biomarker, scientists need to take a liver biopsy from children, which they try to avoid in clinical trials.
“We know what this disease looks like in children, and we know what it looks like in adults,” said Miller, “but as the children grow into teenagers that become young adults and adults, what are the changes in liver biomarkers that are happening over time?”
The study suggests that long-term longitudinal studies and systematically collected real-world data are needed to better understand NAFLD’s natural history in children compared to adults. The research also indicates that, in clinical trials, more histologic criteria should be considered in identifying risk factors in pediatric NAFLD.
The Liver Forum’s Pediatric Working Group is a subset organization of Forum for Collaborative Research. The working group includes scientists and experts from academia, pharmaceutical companies, patients, and regulatory organizations such as the United States’ FDA and European Medicines Agency.
“It’s not a binding guideline,” said Miller, “but it definitely is the best minds coming together to come up with the best plan.”