Scientists at the College of California San Diego Institute of Medication have driven a review to inspect a potential new treatment choice for patients with non-alcoholic steatohepatitis (NASH)-related fibrosis.
A drug that mimics a hormone in the body improved both liver fibrosis, or scarring of the liver, and liver inflammation in patients with NASH, according to the findings, which were published in the online edition of The New England Journal of Medicine on June 24, 2023.
According to Rohit Loomba, MD, the study’s first author and chief of the Division of Gastroenterology and Hepatology at UC San Diego School of Medicine, “Identifying an effective drug for NASH is extremely promising for patients, as currently there are no FDA-approved therapies for this condition.” Patients with NASH may experience deteriorating quality of life and progress to cirrhosis. Its complications may result in liver transplantation or death. Our findings could lead to a new treatment option for NASH-related fibrosis sufferers and advance the science of this disease.
“The study’s findings show that the new potential treatment not only improves fibrosis, but also inflammation and liver injury, as well as significant improvements in multiple non-invasive biomarkers of NASH activity and scarring,”
Loomba, gastroenterologist and hepatologist at UC San Diego Health.
Pegozafermin, a drug, mimicked fibroblast growth factor 21 (FGF21), a hormone that is naturally produced in the body and is secreted by the liver.
The liver’s lipid metabolism and the body’s use of energy are both regulated by FGF21. It has additionally been shown in past examinations to bring down blood glucose and insulin levels, diminishing body weight and liver fat.
According to Loomba, a gastroenterologist and hepatologist at UC San Diego Health, “the study’s results show that the new potential treatment not only improves fibrosis but also improves inflammation and liver injury, along with significant improvements across multiple non-invasive biomarkers of NASH activity and scarring.”
222 individuals with NASH participated in the 24-week randomized clinical trial and were assigned to either the drug or a placebo. Compared to 7% of patients who received the placebo, approximately 27% of those who received the drug at a higher dose showed an improvement in liver fibrosis. The drug’s most common side effects, including nausea, were gastrointestinal in nature.
For the treatment of NASH, a type of nonalcoholic fatty liver disease (NAFLD), there are no medications currently on the market that have been granted approval by the Food and Drug Administration of the United States.
NAFLD affects approximately 24% of adults in the United States, and NASH affects approximately 6%.
With few to no symptoms, NAFLD and NASH are considered silent diseases. Notwithstanding, people who are overweight, have type 2 diabetes, or have a relative with NAFLD are at a higher risk of fostering the illness. NAFLD, which is an umbrella term for a scope of liver circumstances influencing individuals who drink practically no liquor, can prompt cirrhosis, malignant liver growth, and liver disappointment.
Loomba adds that a larger, international, multi-center trial with a more diverse patient population and a longer treatment period will be the next step in this research to better assess the drug’s safety.
Loomba stated, “This drug could be used to treat millions of patients with NASH, including our patients at UC San Diego Health, if successfully shown to be both safe and effective in a larger Phase 3 trial.”
More information: Rohit Loomba et al, Randomized, Controlled Trial of the FGF21 Analogue Pegozafermin in NASH, New England Journal of Medicine (2023). DOI: 10.1056/NEJMoa2304286
