Sharing the Patient Experience in Clinical Trials Will Be the Key to Understanding and Developing Treatments for PSC

In an interview, Dr. Ziad H. Younes of GastroOne highlighted the complexity of treating primary sclerosing cholangitis and some of the experimental treatments under development.
Dr. Ziad H. Younes
Dr. Ziad H. Younes
Gastroenterologist and Director of Clinical Research

Dr. Ziad H. Younes has studied inflammatory bowel diseases for decades and completed a GI fellowship at a combined program between Johns Hopkins Hospital and Johns Hopkins Bayview. Dr. Younes currently works as a gastroenterologist and director of clinical research at Gastro One, working across many GI disorders, including primary sclerosing cholangitis.

Primary sclerosing cholangitis (PSC) is a rare, chronic liver disease that inflames the bile ducts, causing them to scar over time. The bile ducts transport bile, a digestive liquid, from the liver to the gallbladder and the small intestine. The scarring can make it tough for bile to flow, and it can get backed up into the liver, causing damage over time. Patients with PSC are also at increased risk for colon cancer, making annual colonoscopy screenings essential. As PSC progresses it can lead to cancer of the liver cells and cancer of the bile duct. So, it is important to closely monitor patients with PSC.

Unfortunately, there are very few treatments available to PSC patients, and patients with the condition may require a liver transplant to survive. Although a liver transplant can be successful, PSC can re-occur in patients who undergo this procedure, according to Dr. Younes. Because of this, Dr. Younes noted that antibiotics can be used to treat infections in people with PSC. He also shared that a procedure, called endoscopic dilation, can be used to expand patients' bile ducts when they narrow due to scarring.

With very limited treatment options available, clinical trials can help researchers better diagnose, track, and treat PSC. However, clinical researchers don't fully understand the cause of the condition and PSC progresses differently from patient to patient. When asked about the difficulty in diagnosing and treating PSC patients, Dr. Younes discussed that people with PSC may also have other conditions making a diagnosis even more difficult. Currently, many of the active clinical trials for PSC seek to better understand the disease and depend on patients sharing their experienceliving with the condition. Other studies use medical imaging to track how the disease impacts the body.  

But there may be hope with looking at treatments for other GI disorders. People with PSC tend to have other inflammatory bowel diseases such as ulcerative colitis or Crohn’s disease. He mentioned that in his tenure, Dr. Younes has seen several treatment options emerge for Crohn's disease and ulcerative colitis that could be beneficial for PSC.  

There are several clinical trials in clinical development, says Dr. Younes, evaluating the effectiveness of drugs used to treat UC and Crohn’s for patients with PSC. One phase two study, for example, is evaluating sulfasalazine, a drug that is currently used to treat UC. Another study of interest to PSC patients is for elafibrinor, a drug in a phase two study as well. Another, vancomycin, is an antibiotic that fights bacterial infections in the intestines.  

With ongoing research and studies aimed to provide effective treatments for PSC, patient perspectives and close monitoring of them are essential to unraveling researchers understanding of this complex condition. Taking part in clinical trials seems to be the best way to improve knowledge of the disease and support researchers in developing current treatments that slow PSC’s progression, and, hopefully one day, treat PSC all together.

Investigator Bio
Dr. Ziad H. Younes

Gastroenterologist and Director of Clinical Research at Gastro One