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Tackling a Challenging Disease

Primary sclerosing cholangitis (PSC) occurs when the ducts that transport bile become inflamed and scarred. As scarring grows worse, the bile ducts gradually narrow or become blocked. This causes bile to build up in the liver, damaging this vital organ. As PSC progresses, it can cause liver failure.

PSC affects over 30,000 Americans. It occurs most often in men, and usually develops between ages 30 and 50. Symptoms of PSC may include itchy skin, fatigue, abdominal pain, weight loss, jaundice, chills, and fever. Current treatments can alleviate symptoms, but do not slow or stop the disease itself. If liver failure develops, a liver transplant may be necessary.

The cause of PSC is unknown. Scientists believe that bacteria may trigger an abnormal immune response that causes inflammation in the bile ducts. Patients who develop PSC may also have a genetic predisposition to develop the disease. But it has been challenging to identify specific biological culprits and to determine how they interact in this complex disease process. Related diseases may provide important clues. As many as 90 percent of patients with PSC also have inflammatory bowel disease (IBD), typically ulcerative colitis. While the relationship between PSC and IBD is not entirely clear, the fact that they commonly overlap allows researchers at the Resnek Family Center to study them in parallel.

This opens up a number of possible research avenues that could impact both illnesses. Breakthroughs made in one condition could be applied to the other, and vice versa.

The generous philanthropic support of the Resnek family has enabled Dr. Korzenik and his team to accelerate and intensify their search for the fundamental drivers of PSC and related diseases such as ulcerative colitis. This will enable us to define novel biological targets and evaluate new therapies that have the potential to transform treatment for PSC.

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