Novel Antisense Drug from Ionis Shows Promise in Slowing Non-Alcoholic Fatty Liver Disease (NAFLD), Which Affects Approximately One Quarter of US Population; In Phase II Trial, Treatment Inhibited Key Enzyme, Resulting in Slower Progression to NASH

Using a first-of-its-class drug in a clinical trial, an international research effort, headed by a scientist at University of California San Diego School of Medicine, reports that inhibition of a key enzyme safely and effectively improved the health of persons with non-alcoholic fatty liver disease (NAFLD), a chronic metabolic disorder that affects hundreds of millions of people worldwide. The gene silencing approach represents a novel way to reverse NAFLD. The findings were published online on June 15, 2020 in The Lancet Gastroenterology and Hepatology. The article is titled “Novel Antisense Inhibition of Diacylglycerol O-Acyltransferase 2 for Treatment of Non-Alcoholic Fatty Liver Disease: A Multicentre, Double-Blind, Randomised, Placebo-Controlled Phase 2 Trial.” NAFLD occurs when fat accumulates in liver cells due to causes other than excessive alcohol intake. The precise cause is not known, but diet and genetics are believed to play substantial roles. The condition is typically not noticed until the disease is well-advanced, and perhaps has transitioned to non-alcoholic steatohepatitis (NASH), a progressive form that can lead to cirrhosis, liver cancer, and liver failure. There is no cure for NAFLD. Treatment primarily consists of ameliorating contributory factors, such as losing weight, improving diet, exercising more, and controlling for other conditions, such as diabetes and hypertension. No FDA-approved medications exist. In the worst cases, a liver transplant may be required.
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