Panelists discuss how the main treatment goals for patients with primary biliary cholangitis (PBC) include improving liver biochemistry, preventing disease progression to cirrhosis, managing symptoms, and enhancing overall quality of life through regular monitoring and appropriate therapeutic interventions.
A predictive tool that incorporates several blood biomarkers may help to identify primary biliary cholangitis (PBC) patients who are less likely to respond to treatment with ursodeoxycholic acid, known as UDCA, during the early stages of the disease, a study has found.
In recognition of the US Food and Drug Administration accelerated approval of Gilead’s seladelpar (Livdelzi) and other recent evolutions in primary biliary cholangitis (PBC), the latest HCPLive Special Report spotlights a conversation between a trio of subject matter experts on the latest advancements in PBC and their implications for disease management.
At our conference dinner on Friday, April 12th, we were pleased to honor three outstanding individuals who have been with us since the early years of our organization – Doctors Keith Lindor, John Vierling and Eric Gershwin.
Keith Lindor, M.D. was a major leader in the clinical trials for ursodiol, which ultimately became an FDA approved treatment for PBC. He continued to work in the PBC field, training so many physicians who are in the PBC field today. Dr. Lindor has continued to publish on PBC, and has spoken at many of our conferences over the years.
John Vierling, M.D. is another long-time supporter of our organization. Like Dr. Lindor, he has a long list of PBC-related publications to his credit and has presented at many conferences, including the 2024 conference. He has always made himself available when we ask for an explanation of any new developments or to answer a patient’s question.
Eric Gershwin, M.D. is well-known for his PBC research at UC Davis. One of his most important milestones was the development of the mouse model with PBC, which he provides to PBC researchers at no cost. This mouse model has made possible much of today’s PBC research. Dr. Gershwin has the distinction of presenting at every PBC conference but one (and he would have been there but for a travel glitch ).
Given how much our honorees have contributed to PBC research and PBC patients, it was difficult to decide what we could do to express our gratitude. Somehow, a plaque didn’t seem like enough. So we reached for the stars!
When the PBCers celebrated our 10th anniversary in 2006, we honored Linie Moore by naming a star for her. After some thought, we realized that this would be an excellent gift for our honorees, each a star in his field. We purchased a star for each doctor in the same area as Linie’s star, a constellation called Draco next to the scoop of the Big Dipper, which is the prominent part of the Ursa Major constellation. Ursa Major is Latin for “Great Bear”, making this a very appropriate location considering that the connection to our medication URSO is synthetic bear bile!
In the star chart above, you can see where these stars are located. In addition, we have purchased a fifth star representing the PBC Community to honor all those affected by PBC – current patients and those who have lost their battle, as well as families and friends. When Linie created this organization, she wanted us to “never feel alone.” So any time you may be having a bad day, have lost someone to PBC, or just need a little something, look up in the sky and remember that “you are not alone” – there is a whole PBC community, always there.
PBCers, sponsors and speakers gathered in Phoenix from April 11-13, 2024, for our 16th Patient Education Conference. With the theme of Empowering Connections in PBC, 90 members of the PBCer community shared stories, learned from PBC experts, and supported one another over the course of three jam-packed days.
Thursday evening’s registration and reception was a chance to meet and greet, complete with hors d’oeuvres and swag bags. Some participants opted to create bracelets stamped with their “word,” inspired by our “My Intent” online program.
Friday morning opened with a presentation by conference regular Dr. John Vierling on “Diagnosis and Treatment of PBC.” Other presentations included:
After lunch, patients were invited to write stories, while friends and family met for a discussion on caregiving with Lorraine Steihl, CEO of the American Liver Foundation and caregiver to her husband for many years.
The afternoon session featured these programs:
At our conference dinner on Friday, we were pleased to honor three outstanding individuals who have been with us since the early years of our organization – Doctors Keith Lindor, John Vierling and Eric Gershwin. Each was honored with a star naming.
Saturday morning started with three presentations:
Clinical Trials was the post-lunch topic.
Dr. Marlyn Mayo gave the closing presentation on “Managing PBC Itch: Let’s Start From Scratch” – a topic of great interest to many patients. (Dr. Mayo flew in from Dallas to present on her first day in a walking cast after breaking her foot – that is dedication!) Her presentation covered the variety of treatments that help or don’t, and spoke about treatments in trials and how they work. Her talk provided hope that there will soon be new ways to deal with this terrible symptom.
Sponsors
We thank our generous sponsors for helping to make our 2024 conference a success! They were on hand to answer questions, and also enjoyed meeting and learning from patients. Listening to your varied stories gives them a better idea of what patients live with every day and inspires them in their work.
GOLD SPONSORS:
CymaBay Therapeutics
Intercept Pharmaceuticals
Ipsen
SILVER SPONSOR:
GSK
BRONZE SPONSORS:
Calliditas Therapeutics
Escient Pharmaceuticals
Mirum Pharmaceuticals
TRAVEL SCHOLARSHIPS
Array Insights
Mirum Pharmaceuticals
WHOVA APP SPONSOR:
American Liver Foundation
Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”) today announced that the Phase 2b TRANSFORM trial met its primary endpoint, showing statistically significant improvement in ALP (Alkaline Phosphatase) for both doses tested versus placebo. The trial evaluated setanaxib, a NOX enzyme inhibitor, in patients with primary biliary cholangitis (PBC) and elevated liver stiffness.
Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Livdelzi® (seladelpar) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. The use of Livdelzi is not recommended for people who have or develop decompensated cirrhosis.
On June 28, 2024 the Committee for Medicinal Products for Human Use (CHMP), issued an opinion to the European Commission to revoke the marketing authorization for OCALIVA in Europe.
