An extra special thank you to those who created a personalized "giving group" for the campaign and took on their own version of the challenge of 83!

International #PSC Awareness Day is October 29th. How can you get involved? We're glad you asked! Grab your phone and record a very short video wishing folks a "Happy Global PSC Awareness Day". Include your city/province/country. Send your video to us at contactus@pscpartners.org, and we'll post it on the big day. Thanks for helping us raise awareness!

Let's join our global PSC friends to show the world that PSC is everywhere. Watch the 2021 Global Awareness Day video for a smile and inspiration!

One of the recurring themes across the Loon Duct research spotlights is that those who choose to take a deeper dive into PSC often think outside the box looking for ways to maximize the data they can access.

Dr. Mamatha Bhat’s work exemplifies innovative thinking by using artificial intelligence and bioinformatics. Artificial intelligence is a field that leverages patterns in data and interrelationships and allows researchers to better understand a huge amount of complicated data.

Dr. Bhat is a Hepatologist and Clinician-Scientist at the Ajmera Transplant Program in Toronto. As early as her years in medical school at McGill University and then during her residency training, she was particularly interested in the physiology of the liver and liver disease. She was struck by how a liver transplant could, in some cases, “give a patient a second lease on life.”

She relocated to Toronto, where she finished her PhD and started working as a Hepatologist in the transplant program at UHN. She established her own research program in 2019. PSC wasn’t her initial focus, but gradually through years of clinical work and research, she noticed inequities that exist for some liver patients on the transplant waiting list. Patients with PSC and PBC (Primary Biliary Cirrhosis) are among the groups particularly disadvantaged by the existing ranking system.

Inequities on the Transplant Waitlist As many PSC patients know, the MELD (Model for End-stage Liver Disease) score helps determine how urgently a patient needs a transplant. MELD was refined in 2016 to include sodium levels, so you may also see it referred to as MELD Sodium. In theory, this method was meant to equalize the ranking process by evaluating blood tests alone. However, it was created at a time when the predominant disease showing up on the transplant waitlist was Hepatitis C—and so the methodology was designed largely with that disease in mind.

As PSC patients often hear, our disease doesn’t fit inside the box. Blood tests for PSC patients don’t fully represent how sick a patient actually is. Women are further disadvantaged because of the creatinine blood test. For women, creatinine is not a good reflection of their kidney function, because women in general will have lower muscle mass - and creatinine levels correlate with muscle mass. Women are often physically smaller or shorter, and deceased organs more commonly come from men. Furthermore, since women are physically smaller, they may be competing for the same organs as children, and thus ranked further down the priority list.

Dr. Bhat listed a number of other factors that clearly illustrate issues that PSC patients are up against with the MELD score. One size definitely does not fit all.

Diverse Expertise to Solve a Complex Problem Dr. Bhat is looking for ways to understand the full experience of a patient to address some of these inequities. Dr. Bhat’s research interdisciplinary group combines the talents of those with experience in computer science, molecular biology, bioinformatics, nanomedicine, and clinical medicine. This approach brings a diversity of thought to the table, and complements Dr. Bhat’s interdisciplinary training background. Dr. Bhat noted that she has collaborated with PSC expert Dr. Gideon Hirschfield on this project.

By bringing different perspectives together, it allows new questions to be asked and new approaches to be considered. Tools of artificial intelligence can leverage the changes in a patient’s status over time instead of ranking a patient’s sickness based on a small number of blood tests captured on a single date.

The uncertain nature of how PSC progresses is a huge stressor for patients. Artificial intelligence can help to understand how a patient’s disease progresses over time using a greater number of variables—and potentially how to better predict its trajectory going forward.

Imagine reporting in for an annual PSC checkup, and being able to have a clearer idea of how your disease is progressing based not only on your own history but the combined data of hundreds or thousands of patients over time. Then combining other variables like where you live and the patterns of deceased organ availability come into play. PSC asks a lot of patients to navigate uncertainty, but there is also hope knowing that innovative research like this is being applied to our rare disease.

by Kristian Stephens

A new international study offers support for increasing the use of living donor liver transplantation (LDLT) in Western countries to help meet the demand for liver transplants. This study titled “Low utilization of adult-to-adult LDLT in Western Countries despite excellent outcomes: International multicenter analysis of the US, the UK, and Canada.” is reported in the Journal of Hepatology.

The one-, five-, and 10-year patient survival rates were 92.6%, 82.8%, and 70.0% in the USA; 96.1%, 89.9%, and 82.2% in Canada; and 91.4%, 85.4%, and 66.7% in the UK, respectively in this study. Therefore, continued efforts to increase LDLT usage in these countries may be warranted due to the growing waitlist and differences in allocation that may disadvantage patients currently awaiting liver transplantation.

If you are interested in getting involved, please send us an email at: ContactUs@PSCPartners.ca

A Billion $ Canadian Government Commitment

In 2019, the Canadian government committed $1 billion to setting up Canada’s Rare Disease Drug Strategy with the goal of providing access to effective therapies for persons living with rare diseases. This opportunity comes at a time when transformative and life-saving therapies are emerging as the first effective treatments for rare and ultra-rare patients with progressive, severe or life-threatening conditions.

CORD, the Canadian Organization for Rare Disorders, is hosting the CORD 2022 Fall Conference Nov 21-22 in Toronto. This two-day conference will bring together experts and participants that will engage in strategic planning sessions to arrive at proposals for key components of the Canadian government's Rare Disease Drug Strategy. Patients are invited and can attend in-person or virtually (starting at $50).

How is this relevant to PSC? Treatments currently in the development pipeline for PSC may prove effective, may at some point be approved for use in PSC in Canada, and will need to be priced in a way that reduces financial barriers to access treatment. Please consider attending this conference if you have an interest in national health care investments and policies. This conference is an opportunity to add your voice to ensure that the needs of the Canadian PSC community are included in these proposals coming from the rare disease community. Register here.

Entitled From Vision to Reality: Canada’s Rare Disease Drug Strategy - Transforming Commitment to Investment in Optimal Patient Outcomes, the conference plans to develop demonstration projects that can serve as “proof of concepts” for the key elements of an essential Rare Disease Drug Program to support effective and cost-effective use of rare disease therapies.

CORD's proposal: Canada can demonstrate global leadership by implementing a highly effective, cost-effective, and sustainable Rare Disease Drug Program built upon three pillars:

from the European and American Medical Liver Societies

Want to share your story? Email us! ContactUs@PSCPartners.ca

Upcoming PSC Peer Group Zoom Rooms!

Patients with PSC and Post-Transplant 

► Register Here

For Those Living with Loss 

Spouses, Caregivers, & Parents 

Watch the PSC Partners website, newsletter, and social media for updates.

PSC Partners Canada uses Facebook regularly to share news of interest to the Canadian PSC community. If you are not a Facebook user, the feed of postings is shared to a page on our website at pscpartners.ca. The Loon Duct is sent quarterly, so if you are wishing for more frequent contact, remember to follow us on Facebook, or check in periodically on the website.

(647) 848-6953 | ContactUs@PSCPartners.ca | PSCPartners.ca