The new therapy strategy for primary sclerosing cholangitis, a liver disease that at present still cannot be cured with medication, shows initial measureable success with the nor-ursodeoxycholic acid (norUrso). A multi-centric phase II study with 45 involved centres from all across Europe led by MedUni Wien, demonstrated that when norUrso was administered to 161 patients for the first time "there were significant improvements in liver function test results in all tested dosing" according to Michael Trauner, head of the clinical department for Gastroenterology and Hepatology at the MedUni Vienna. The results have now been presented by Trauner in the Late Breaker plenary session at the largest international liver congress (EASL) with more than 10 000 participants in Barcelona.
"This is an extremely promising result. This means that a phase III study can now be initiated, which also examines the long-term effects, the progression of the disease", state hepatology experts at the MedUni Vienna. Trauner's presentation was one of four lectures which was explicitly distinguished by the EASL congress (www.easl.eu/) itself last weekend, from the many lectures during the press conference. Generally the MedUni Vienna, which is one of the world's leading centres for the research of cholangitis and other liver and gall bladder diseases, was present with a multitude of lectures that featured prominently and were the best attended: Philipp Schwabl was awarded the prize for the best congress presentation in the area of fundamental research for his work on new mechanisms in the treatment of portal hypertension and liver fibrosis.
About primary sclerosing cholangitis
Primary sclerosing cholangitis (PSC), is a liver disease which at present still cannot be cured and mainly affects younger people, especially men between the age of 30 and 40. It is considered to be a rare disease, which is mainly discovered by chance or in the course of jaundice and is characterised by disrupted bile production. The disease is typically associated with inflammatory bowel disease with a high risk of colon cancer. In its course this disease can develop into liver cirrhosis and bile duct cancer. Its cause is not yet known but it is suspected to be linked to inflammation in the bowel.
Preliminary studies for these multi-centric studies now being presented to patients with PSC, following years of previous research on the modes of action in pre-clinical animal models led by Trauner, demonstrated that norUrso (nor-ursodeoxycholic acid) has a direct impact on the bile duct and toxic substances, the result of an increase in bicarbonate-enriched bile flow, are flushed from the biliary tract.
In Michael Trauner's team Emina Halilbasic at the clinical department for Gastroenterology and Hepatology of the University Hospital for Internal Medicine III of MedUni Vienna is researching this treatment strategy in particular in the clinic and laboratory. It could be clearly demonstrated that an improvement in the bicarbonate secretion in the bile flow can reduce or prevent inflammation and that bicarbonate anions act like a protective screen for bile duct cells. Also norUrso has immediate inflammation and fibrosis-inhibiting effects which are currently being researched further.
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