Sclerosing cholangitis Previously incurable liver disease from now on treatable?

Scientists report first therapeutic success with new bile acid
The primary sclerosing cholangitis mainly affects younger people between the ages of 30 and 40 years and is a currently incurable liver disease. In a recent European multicentre phase II study under the direction of the MedUni Vienna and in close cooperation with the Medical University of Graz and the Hannover Medical School (MHH) is now the first time a cure of the disease using a synthetically produced bile acid (active ingredient nor-ursodeoxycholic acid / Nor-Urso) succeeded.

So far, the primary sclerosing cholangitis remained one of the last major "black boxes" in hepatology, "one of the last unresolved puzzles," said study director Michael Trauner, Head of the Clinical Department of Gastroenterology and Hepatology at MedUni Vienna in a press release on the study results. If healing on the basis of the artificial bile acid "Nor-Urso succeeds, that would be a remarkable breakthrough in hepatology," stresses Trauner. The researchers published their results in the journal "Journal of Hepatology".

With the help of synthetic bile acid, scientists have successfully treated the primary sclerosing cholangitis - a previously incurable liver disease. (Image: magicmine / fotolia.com)

Incurable liver disease with poor prognosis
The current study was based on data from 161 patients with primary sclerosing cholangitis from 45 treatment centers in 12 countries (Austria, Germany, Belgium, Denmark, United Kingdom, Hungary, Lithuania, Holland, Norway, Spain, Finland and Sweden). So far, the prognosis for incurable rare liver disease is very poor, as it can develop in the course of liver cirrhosis and bile duct cancer. The disease is characterized by a disruption of bile production and affects 0.01 percent of the population, according to the MedUni Vienna.

Significant improvements in liver enzymes
In their latest study, the scientists are testing the use of synthetic bile acid in the treatment of primary sclerosing cholangitis - with success. "There were significant improvements in liver function in all doses tested," reports study leader Trauner. This is a promising result. "It is now possible to start a Phase III study that also looks at the long-term effects on disease progression," continues Trauner.

Synthetic bile acid clinically effective, safe and well tolerated
According to the researchers, Nor-Urso was clinically effective, safe and well tolerated in the study. The synthetic bile acid acts directly on the bile duct and flushes the bile ducts by increasing the bicarbonate-rich bile flow of toxins. "The active ingredient circles between bile and liver and acts exactly where the inflammatory processes take place, namely directly in the bile duct," emphasizes the study leader.

Phase III study planned
According to the researchers, current results suggest that Nor-Urso may also help to stop liver fibrosis and improve connective tissue. This should now be investigated in a phase III clinical trial. This will take around three years and, if the result is positive, treatment of the hitherto incurable primary sclerosing cholangitis with synthetic bile acids could take place in the future. (Fp)