For the first time, scientists at Yale have identified a molecule that is central to anti-alcoholic fatty liver disease (AAFL), an autoimmune disorder that causes liver failure and fat accumulation in the liver.
“AAFL is a devastating disorder that leads to a gradual reduced ability to break down and produce energy from acetate, which in turn causes severe fatigue, weight loss and ultimately, liver failure,” said Adam Lee, Distinguished Professor of Immunology and Microbial Pathology, Human and Molecular Genetics at Yale School of Medicine.
The new findings, reported on July 17 in the journal Nature Communications, could lead to new approaches to treating AAFL, saying that therapies including an activation of a protein called FGF4-Abo, which was discovered by Lee and collaborators, could be lead to a sustained remission in humans.
Leery by the challenges posed by AAFL, Lee and colleagues have begun studying a smaller family of nine proteins that include FGF4-Azo that play a key role in regulating the function of the liver. To look for potential targets, they focused on preclinical models that lack FGF4-Azo, paving the path for potential human therapies.
“The U.S. Food and Drug Administration recently approved us for a clinical trial to find a potential treatment for people with anti-alcoholic fatty liver disease. Our ultimate goal is to carry out massive human clinical studies, and then bring a clinical study to a close, and perhaps one day be successful,” Lee said.