According to a press release from the German-American biotechnology company Immunic Therapeutics, the Company recently began a proof-of-concept trial for its investigational primary sclerosing cholangitis treatment, IMU-838.
About Primary Sclerosing Cholangitis
Primary sclerosing cholangitis (PSC) is a rare, progressive condition characterized by the inflammation and eventual scarring of bile ducts in the liver. Bile ducts are small tubes that run through the liver and connect it to the pancreas and small intestine, allowing for bile (a digestive fluid secreted by the liver) to reach the small intestine to aid in the digestion of certain fats.
As the bile ducts scar, obstructions may form in the pathway, trapping bile in the liver and potentially causing serious damage. The condition is associated with an increased risk of bile duct cancer, repeated and persistent bile duct infection, and cirrhosis with eventual loss of liver function.
Interestingly, physicians and researchers haven’t established any particular cause for PSC. Like many other conditions with “unknown” singular causes, the prevailing belief is that PSC results from a combination of certain genetic and environmental factors. Some theorize that PSC is an erroneous immune response to the presence of some toxin or infection.
About IMU-838 and its Proof of Concept Trial
IMU-838 is an orally-administered small molecule inhibitor. Immunic researchers believe the drug will inhibit the activity of a gene called DHODH, which is believed to play a role in the function of immune reaction mediators called Th17 cells. These cells play a role in encouraging inflammatory immune responses, and there is some evidence that suggests that the deregulation of body Th17 levels plays a significant role in the development of certain autoimmune conditions.
It seems that researchers, if not assuming that PSC is an autoimmune disease, may be at least taking steps to evaluate and limit any present immune response in PSC patients.
The new open-label, proof-of-concept study will be conducted with 30 participants who have received PSC diagnoses. For six months, patients will take 30mg of IMU-838 daily. At the end of this period, participants will have their serum alkaline phosphatase levels evaluated. Since changes in serum alkaline phosphatase are associated with the progression of PSC, small changes or lack of change would suggest a positive result in the trial.
Immunic is testing IMU-838 for a number of autoimmune conditions. Phase 2 studies of the drug are already being run in patients with relapsing-remitting multiple sclerosis and ulcerative colitis, two other autoimmune conditions. If anything, IMU-838’s potential as a PSC drug is a relatively new concept.
The average period of time from diagnoses of PSC to death or liver transplant is only 15 years. Why is it important to develop possible treatments for a condition without necessarily knowing the cause? Share your thoughts with Patient Worthy!
