Ana Lleo, MD, PhD: Primary Sclerosing Cholangitis Disease Biomarkers

Ana Lleo, MD, PhD, professor of internal medicine at Humanitas University, sat down with HCPLive to discuss key takeaways from her presentation about primary sclerosing cholangitis (PSC), our current understanding of disease biomarkers, and areas where further research may be warranted.

Lleo was a speaker during the session “Advances in the Management of Primary Biliary Cholangitis (PBC), Primary Sclerosing Cholangitis (PSC) and Cholangiocarcinoma: A Global Perspective” at The Liver Meeting 2023 from the American Association for the Study of Liver Diseases (AASLD) in Boston this weekend, where she presented on PSC disease biomarkers.

Speakers covered topics ranging from unmet clinical needs surrounding ethnicity and gender to diagnosing cholangiocarcinoma. Lleo’s portion of the session focused on biomarkers for PSC, highlighting the need for new drugs and better structure surrounding clinical trials.

“It's difficult to discern the clinical trials because you don't really know exactly the endpoint that fits best. Each one of them has some limitations, even the old alkaline phosphatase that we have been using for a long time is not ideal in PSC because there is a lot of variability among patients. Even within the same patient, different regions of the liver present with different characteristics,” Lleo explained.

Although PSC does not have many unmet needs, Lleo cited the diagnosis of cholangiocarcinoma and the need for effective therapy as the most prevalent among current shortcomings surrounding PSC. She further referenced a lack of available PSC biomarkers considered “a good surrogate of the disease outcome,” highlighting a need for development in this area.

“We have some biochemical biomarkers like alkaline phosphatase, bilirubin, and we have and we use some biomarkers that establish the level of fibrosis. For instance, transient elastography or liver biopsy, and they do have a role there. And then there is a lot and I think we will see a lot on imaging, mostly MRI,” Lleo said, pointing out the need for specialized radiologists for imaging-based approaches that may limit their practicality in clinical practice. “I think there is a lot of work there. There is a specific research group on MRI and PSC and I think we will probably see a lot of development there.”

Looking ahead to the future of PSC research, Lleo acknowledged there is still a lot that remains unknown about PSC: “We don’t know much about the role of the immune system, the gut-liver axis connection, the role of microbiome. But what I would really like to see is a therapy for these patients. I think that's what we all want,” Lleo concluded.