Primary Sclerosing Cholangitis and the Push for Treatment Options

Primary sclerosing cholangitis (PSC) is a rare but serious disease that can affect people living with inflammatory bowel disease (IBD). No treatments currently exist for this condition, but researchers and advocacy groups are working to change that by building awareness and working to develop new therapies.

What is PSC?

To understand what PSC is, it helps to understand a little bit about the liver and bile ducts. The liver is the largest internal organ in the body. It performs more than 500 vital functions to help keep your body healthy. One of the liver’s jobs is to make bile (digestive liquid). Bile is important for absorbing fats and vitamins A, D, E, and K. The bile ducts carry bile to the small intestine. When a person has PSC, inflammation prevents bile from flowing to the intestines. The bile stays in the liver, damaging liver cells. This can lead to scarring (sclerosis) and, possibly, cirrhosis.

The link between IBD and PSC

PSC affects about 30,000 people in the U.S. However, PSC often occurs alongside IBD.

• Between 70% and 90% of people with PSC also have IBD, most often ulcerative colitis (UC).
• About 5% of people with IBD develop PSC.

Researchers do not yet fully understand why PSC and IBD occur together. They are studying genetics, the immune system, the gut microbiome, and environmental factors as possible contributors.

A high symptom burden and serious risks

PSC can be difficult to diagnose. Many people have no symptoms at first. They learn they have PSC after routine blood work or imaging tests. As the disease progresses, symptoms often appear and can have a major impact on daily life.

Common symptoms include:

• Intense itching (pruritus)
• Pain in the upper right abdomen
• Severe fatigue
• Yellow skin and eyes (jaundice)

Ongoing inflammation and bile duct damage from PSC can raise the risk of certain cancers, including:

• Bile duct cancer (cholangiocarcinoma)
• Colon cancer
• Gallbladder cancer

Because of these risks, people with PSC require close, ongoing monitoring by their care team,
 

A major unmet need

Right now, doctors do not have an approved treatment to slow or stop PSC. Care teams focus on managing symptoms and monitoring for complications, including regular liver checks and watching for problems early. If PSC becomes severe, some people need a liver transplant. In rare cases, PSC may come back after transplant.

That gap between what people need and what medicine can offer right now puts many families in “wait and watch” mode. This makes urgent PSC research and greater awareness more important than ever.

Research to accelerate scientific discovery and find treatments

PSC Partners Seeking a Cure, a U.S.-based advocacy organization, is working to improve understanding of PSC. The group hosts an annual patient conference that brings together patients, caregivers, family members, clinicians, and researchers to share information and drive progress.

In advance of their annual conference in September 2025, PSC Partners, along with the Resnek Family Center for PSC Research, organized a meeting for experts in hepatology and gastroenterology.

There, researchers shared emerging findings and discussed the most urgent questions in PSC-IBD research. A public report from the meeting will be available in the near future.

What is the Resnek Family Center?

The Resnek Family Center is located at Brigham and Women’s Hospital in Boston. Its primary goal is to develop effective therapies for PSC. The center is taking multiple approaches to move research forward. Current efforts include:

• Exploring dietary interventions that may influence disease activity
• Identifying biomarkers that better reflect disease improvement, since current testing methods are limited
• Potentially repurposing existing medications to help shorten the drug development timeline

• Studying the underlying causes of PSC and its connection to IBD

   

Researchers from across the U.S. contribute to the center’s tissue and data repository, which includes bile, blood, stool, and liver tissue samples. This shared resource supports both basic and clinical research and helps accelerate discovery.

We are here to help

You don’t have to navigate PSC or IBD alone. The Crohn’s & Colitis Foundation is here to support you. Contact the IBD Help Center for personal answers from knowledgeable information specialists.

• Call the free helpline (1-888-MY-GUT-PAIN).
• Send an email to [email protected].
• Text chat with a live agent (subject to availability).

Foundation agents can answer your questions and connect you to other helpful resources. The service is available in more than 170 languages.

(c) 2026 Mirum Pharmaceuticals, Inc. All Rights Reserved. US-VOL-2600003 02/2026