The main aim of the clinical studies in primary sclerosing cholangitis (PSC) is to determine effective means of treatment and diagnosis. Challenges are manifold, but the lack of diagnostic markers for PSC and for cholangiocarcinoma in PSC means that both conditions are diagnosed late. The lack of effective medical treatment of PSC means that, unfortunately, PSC is the leading indication for liver transplantation in Norway.
PSC is a slowly progressive disease with a course of one to two decades from the identification of early stages of disease manifestations until end-stage liver disease. This results in difficulties in establishing a robust level of evidence for the benefit of any treatment in delaying liver transplantation. At present, no medical therapy with proven efficacy is available. Several compounds are being investigated in phase I and phase II trials. So far the Norwegian PSC research center participates in the nor-ursodeoxycholic acid trial from the Falk Pharmaceutical company, but other drugs will also be tested. Ongoing studies also aim to determine the optimal mode of endoscopic therapy for narrow strictures (dilatation or stenting).
Another key topic for the clinical research group is to determine biomarkers for disease severity, including markers that can be used to determine the effect of medical treatment. This includes biomarkers in blood and bile, along with evaluation of novel imaging modalities. Patients are also closely followed after liver transplantation to determine factors that contribute to the development of the recurrent disease (occurring in 15-30%).
Establishing diagnostic markers for PSC in patients with inflammatory bowel disease could serve crucial for diagnosing patients before the scarring of the bile ducts has become irreversible. In collaboration with the Inflammatory Bowel Disease in South Eastern Norway (IBSEN) study group, ongoing efforts aim to determine factors that correlate with even sub-clinical bile duct changes on cholangiography in patients with inflammatory bowel disease. Likewise, it is a priority topic to determine biomarkers for pre-clinical cholangiocarcinoma in patients with PSC to allow for effective treatments (see LINK TIL FUNCTIONAL GENOMICS).
All studies within the clinical research groups are performed in close collaboration with the International PSC study group (www.ipscsg.org). Key investigators are clinicians, typically funded by Oslo University Hospital and Haukeland University Hospital. Some dedicated time for research is supported by the Norwegian PSC Research Center.