A trial drug could offer hope for patients with primary sclerosing cholangitis.
A recent study showed that Norursodeoxycholic acid significantly reduced levels of serum alkaline phosphatase in patients with the rare liver disease primary sclerosing cholangitis at 12 weeks.
This enzyme is found in the blood and the levels increase in the presence of liver disease.
A randomized double-blind study enrolled 159 patients with elevated serum alkaline phosphate levels. This study lasted 12 weeks and researchers followed up after 4 weeks.
Patients either received 500-mg, 1000-mg or 1500-mg of Norursodeoxycholic acid daily or a placebo.
The endpoint of the study was the change in serum alkaline phosphatase before and after treatment.
Researchers found that patients receiving 500-mg of Norursodeoxycholic acid experienced a decrease in serum alkaline phosphatase levels of 12.3%. Patients receiving 1000-mg of Norursodeoxycholic acid saw their serum levels drop 17.33%, while patients receiving 1500-mg of Norursodeoxycholic acid saw a decrease of 26%.
Adverse events occurred similarly in all treatment groups and the placebo group.
Severe itching of the skin occurred at low frequencies. There were 7 adverse events in the 500-mg group, 7 events in the 1000-mg group, 12 events in the 1500-mg group, and 10 events in the placebo group.
"This is a rare but life-threatening liver disease affecting mostly young individuals,” Frank Tacke, MD, PhD, EASL governing board member, said in a press release. “Norursodeoxycholic acid provides hope for this population, as this could be the first pharmacological treatment option for this disease.”
Patients with primary sclerosing cholangistis face symptoms such as itching, tiredness, cirrhosis, high blood pressure, liver failure, and liver cancer. Currently, there is no treatment, but researchers are hopeful that one could be developed with further trials.
"Our study demonstrates that Norursodeoxycholic acid could be a viable treatment option for patients with this chronic and debilitating condition," said study lead author Michael Trauner, MD. "Norursodeoxycholic acid provided a safe and effective option for patients with primary sclerosing cholangitis and the treatment warrants further investigation in a larger-scale Phase 3 trial."