Volume 4, Issue 3
March 2023
► Highlights of the Halifax 2023 Canadian Liver Meeting
► Breaking News: Federal Funding for Rare Diseases
► Clinical Trial News: Newly Launched Canadian Sites
► TRIANGLE Training Program
► Year End Fundraising Update
► PSC Patient Spotlight
► UHN Funding from CZI for Patient-Led Research
► Canadian PSCers in the News
► Upcoming Events
Highlights of the Halifax
2023 Canadian Liver Meeting
Photo Credit: Joint Canadian Digestive Diseases Week™ and Canadian Liver Meeting Conference 2023
Canadian PSC patients were well represented at the recent March 3-5 Canadian Liver Meeting in Halifax. This event was held jointly with the Canadian Digestive Disease Week. The Canadian Liver Meeting is hosted by the Canadian Association for the Study of Liver Disease (CASL).

The PSC Partners Canada team attended and distributed materials about the resources available for Canadians living with PSC and about the research grants the organization offers. President Mary Vyas and patient partner Virve Aljas hosted the PSC Partners exhibit booth, attended relevant presentations, and connected with researchers, patient advocates, and industry representatives. Virve represented the patient voice in the inaugural meeting of the TRIANGLE training program (see the TRIANGLE article in this issue for more information). Lapel pins featuring livers, guts, and the PSC Partners Canada logo were popular swag items generously donated by a PSC patient to help us get our word out!

The fourth CASL - PSC Partners Canada Award for the best PSC research abstract was presented to Diana Nakib, from the University of Toronto, for her oral abstract entitled “Examining the Modified Cellular Ecosystems Underlying Inflammation in PSC.” You may have virtually met Diana during the 2022 PSC Partners Annual Conference where she presented a poster on the work she and her colleagues are doing in mapping the cellular landscape of the PSC liver.
Above: CASL Research Committee Co-Chair: Christopher Rose, Diana Nakib, & Mary Vyas at presentation of CASL - PSC Partners Canada 2023 award for best abstract on PSC at the Canadian Liver Meeting.

Right: Toronto Centre for Liver Disease's Gideon Hirschfield visiting the PSC Partners booth and patient partner Virve Aljas
Abstracts from this meeting are published here and are open access. Two studies examining data from Alberta (2008-2019) and Ontario (2002-2021) both showed rising incidence and prevalence of PSC. It is hard to know yet if this is due to improved PSC identification and/or PSC clinical care. 

The Loon Duct staff is preparing layperson summaries of two talks we think Loon Duct subscribers will be interested in: What’s New in PSC: Guidelines and Terminology (Julian Hercun) and a Living Donor Liver Transplantation: 20 Years Experience & Update (Nazia Selzner). Please stay tuned.

1. See O - 018, P - 24, P - 31 and P - 32
Breaking News!
Government of Canada Funds Improved Access to Affordable and Effective Drugs for Rare Diseases
"This investment will enable improved access to new and emerging drugs for Canadians with rare diseases, including children, as well as support to early diagnosis and screening. This is a first step in looking at drugs for rare diseases coverage from a national perspective – consistently ensuring that patients can access the drugs they need regardless of where they live in the country."
-- Adam van Koeverden, Parliamentary Secretary to the Minister of Health and to the Minister of Sport
On March 22, the Honourable Jean-Yves Duclos, Minister of Health, announced measures in support of the first-ever National Strategy for Drugs for Rare Diseases, with an investment of up to $1.5 billion over three years. While PSC does not yet have an approved treatment, the establishment of federal support for the needs of Canadians living with rare disease is good news.

In addition to establishing funding for access to treatments, research and infrastructure will be funded: "$32 million over five years to the Canadian Institutes of Health Research (CIHR) to advance rare disease research with a focus on developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network; and, $16 million over three years to support the establishment of national governance structures, such as a Health Canada secretariat and a stakeholder Implementation Advisory Group, to support the implementation of the Strategy."
Clinical Trial News:
Newly Launched Canadian Sites

Participants in the PSC Partners Patient Registry will be contacted directly by a Registry email if a study sponsor requests Registry support in spreading the news about their studies or about clinical trial participation. Such emails will contain information about how to learn more. The Registry will never release identifying patient information to study sponsors. No identifying information leaves the Registry.

Please note the following new research opportunity for Canadians: The PACIFIC study has recently started enrolling patients at two Canadian sites (Toronto and Vancouver) and will be adding two additional enrolment sites in the near future (Edmonton and Quebec City). See the information below from the study sponsor to learn more.

As an example of how the Registry notices work, in this case, Canadian patients who are enrolled in the Registry and who have indicated that they experience itch (pruritus) and meet other eligibility requirements, such as age (between 18-80), will receive an email with a notice that they may be eligible for this study and and will be provided information about how to learn more. If you are interested in receiving such notices, please join the Registry at www.pscpartnersregistry.org and update your Clinical Survey annually, or whenever your condition or symptoms change. 

From the study sponsor:

The PACIFIC research study is seeking adult participants with moderate to severe itch (pruritus) caused by cholestatic liver diseases like PBC or PSC. The study is evaluating an investigational, once-daily, oral medication that targets and blocks the nerves that trigger cholestatic itch. Learn more and find out if you could be part of this opportunity to develop a potential new treatment option.
Details about PACIFIC study can be found here.
Programs Including the Patient Voice by Design – TRIANGLE Training Program
Written by Virve Aljas
PSC Partners Canada is a shining example of how patients and their supporters can have a positive influence when they become directly involved in research, policy-making, and advocacy. We have a unique perspective to share on the lived experience of this disease, and can help researchers understand that the outcomes of their work are more than theoretical—they could be life-changing for patients.

Many organizations are designing programs to intentionally incorporate the patient’s perspective. One example of this is the TRIANGLE Training program, launched in 2022. I’ve had the pleasure of becoming involved as a patient partner. TRIANGLE stands for “Training a new generation of researchers in Gastroenterology and Liver”. This is a national training program for undergraduate and MD summer students, Ph.D. students, research fellows, and early career faculty aimed at transforming research and patient care in the fields of gastroenterology and liver.

I was invited to be a part of several initiatives as part of the inaugural-year activities, including scoring TRIANGLE applications, and meeting researchers in person at the inaugural TRIANGLE Summit. Dr. Roshane Francis of Crohn’s and Colitis Canada facilitated a session to help the awardees learn how to communicate complex concepts in their work to a variety of audiences, including patients.

The researchers are working on topics across the spectrum of Gastroenterology and Liver diseases, but I was thrilled to see some focus on PSC specifically as a lot of work is happening on areas that affect PSC patients.

Awardee topic highlights:
Dr. Rabia Khan, MD, SickKids in Toronto – Studying the outcomes of children and adults with both Inflammatory bowel disease (IBD) and PSC. This work will develop a way of identifying people with PSC-IBD from within provincial health data, validating an algorithm so that we continue to study this condition in the future using this data. This work also aims to predict which PSC-IBD patients have worse outcomes to try to prevent complications for these patients.

Dr. Ana Teresa Limon Miro, PhD, UAlberta in Edmonton - Understanding and improving the way we evaluate and treat malnutrition in patients with cirrhosis: from research techniques to innovative practical tools. Most patients with cirrhosis are malnourished, which doubles their risk of liver-related complications and death. This research will focus on gathering more information to major gaps clinicians face to diagnose and treat malnutrition for these patients.

Dr. Or Steg Saban, MD, SickKids in Toronto - Children who are waiting for liver transplantation are known to have poor muscle mass and function, a condition known as sarcopenia. Even though pediatric hepatologists are aware of this important complication and address nutrition in patients with advanced liver disease, mechanisms that cause sarcopenia have yet to be studied in these patients. In this study, they will compare muscle tissue from patients with and without chronic advanced liver disease.

Dr. Yasmin Nasser, MD, Ph.D. FRCPC, U of Calgary in Calgary - Patients with IBS experience abdominal pain, changes in bowel habits, and poor quality of life. Yoga is a mind-body intervention that can be effective in treating IBS. Dr. Nasser aims to study whether yoga improves IBS symptoms by changing the gut microbiome.

Emily Johnson, Graduate student—PhD, UAlberta in Edmonton – With a high symptom burden, anxiety and depression often go untreated for patients with cirrhosis, even though they affect 1 in 2 patients. Her research studies the mental health impacts of a 12-week online mental wellness and chronic disease skills program for people living with cirrhosis.

TRIANGLE is co-funded by the Canadian Institutes of Health Research, the Canadian Association of Gastroenterology, Crohn’s and Colitis Canada, the Canadian Association for the Study of the Liver, and the Canadian Children Inflammatory Bowel Disease Network. You can learn more about TRIANGLE at www.triangleprogram.org.
Year End Fundraiser Update

Thank you to all who came forward and donated, doubling their donation thanks to a generous matching grant offer. We raised a total of $19,152 in that campaign, which with the match became $38,304! In December alone, enough was donated to support one year of a typical 2-year PSC research grant. Thank you Canadian donors!!
Stay tuned:

Canada Walk 83.01 will return this summer with a different format. We are considering getting together to walk (and eat!) for those who live in the vicinity of Toronto or Vancouver. We love seeing each other in person so this seems like a great time to do it.
 
Our Online Auction will return this fall. The committee will have their first meeting in March. It isn’t too early to start thinking about connections that you have to businesses, resorts, cabins, restaurants, sport or cultural events. We are hoping to have predominantly gift certificates that can be easily mailed or sent electronically to the winning recipients. Contact Sandy@pscpartners.ca with your ideas! The last virtual auction had an unexpected side benefit of a community experience. We heard from many who participated by soliciting auction items, donating auction items, and participating in the planning and the online bidding, that the event helped us feel connected and supported, in addition to funding research. Let us know if you'd like to get involved!
 
An easy and fun fundraising idea! Are you in a golf league? (This can easily be adapted for a book club or a walking group etc.)

One of our PSC moms asked her league executive if they’d be willing to have a day when members could donate to PSC Partners Seeking a Cure Canada. Promotion of the day only involved sending the members a video about PSC and some basic information. She asked a friend to join her at the course early that day to collect donations and each foursome was given a yellow ball that they tried not to lose during their round. She had a few prizes for yellow balls that were returned. Most players wore yellow that day. The ladies had fun and appreciated being able to support an important cause that affected someone they knew.
 
If anyone is interested in doing this with their group, please contact sandy@pscpartners.ca and she will walk you through the steps to organize it.

Canadian Patient Profile: Hil Hamilton

Article by: Matt Hodgson
Meet Hil Hamilton, a Canadian PSCer in Nova Scotia, who was an early guest on the Sickboy Podcast. Hil’s guest appearance aired all the way back in 2016, which was a year after Hil’s diagnosis and years before podcasting was the powerful medium it is today.

Hil (pronouns they/them) battled just to reach a diagnosis of PSC, an unfortunate reality for many early PSC patients. Hil described a number of barriers that resulted in a delayed diagnosis of PSC. Shortly before experiencing the first set of symptoms, Hil traveled to several destinations where a type of infectious disease could be a possibility. They saw a series of infectious disease doctors before one doctor had a hunch, put in the extra effort, and made the referral to a hepatologist where a liver biopsy finally provided certainty as to what was behind their various symptoms.

Like many newly diagnosed patients, Hil turned to the internet to find out as much as they could about the disease. During this initial search, Hil came across PSC Partners and became engaged with the community-based science and recognizing the importance of getting into the Patient Registry.

Discussing your PSC with someone for the first time can be challenging. In Hil’s experience, “what someone might want to hear is different from what you as a patient want to communicate.” This process can be made more difficult with the dual nature of PSC as a rare and invisible disease.

Talking publicly about PSC early into their journey prepared Hil for future conversations with healthcare providers, friends, and co-workers. “Just the other day, I had a neighbour say to me ‘how were you just in the emergency room two days ago? You look so good.’” This interaction is familiar to Hil, “Sometimes if people can’t see it, they don’t believe it to be true.”

With a Master’s degree in marine biology, Hil is accustomed to communicating in scientific and academic terminology. This background proved useful when communicating with healthcare providers, as they would often have to educate a new provider on the disease.

Meeting new healthcare professionals is a familiar reality for Hil. Over the past couple of years, Hil estimates they’ve gone to the emergency room over 40 times. “A doctor recently came in and said they didn’t know much about PSC but asked what they should know.”

Hil explained that they’ve seen firsthand how using precise language can help a doctor, who may not have encountered PSC before, provide more accurate and timely care. Hil stresses that you don’t want to lie, but sometimes accentuating symptoms (for example, describing symptoms at their worst) can be needed to be taken seriously.

For instance, a few years ago Hil experienced the ill effects of untimely treatment, being hospitalized for 10 days with cholangitis that turned into septicemia which is blood poisoning by bacteria. This is exactly the type of situation Hil hopes others can avoid by advocating for themselves when seeking treatment. 

One resource that Hil recently came across was a printable “PSC Alert Card” from PSC Support, the UK patient organization dedicated to improving the lives of patients with PSC. The card provides accurate and succinct information that an ER physician, or other primary care provider, can quickly review and get up to speed on PSC. PSC Support UK has generously offered to allow PSC Partners Canada to modify the UK Alert Card to make it specific for the Canadian healthcare system, and PSC Partners Canada has been working with Canadian healthcare providers to make these modifications. Stay tuned on how to get your Canadian copy soon! 

Although the original Sickboy Podcast episode is no longer available, Hil credits it for pushing a conversation about PSC, and interacting with the healthcare system with this disease, into the broader discourse. “Most people in my circle of friends learned I had PSC from the episode,” explained Hil, “I hoped others with PSC would find it helpful or useful to hear someone talking about their symptoms and experience.”
Photo provided by Hil Hamilton
Patient-led research will investigate mechanisms behind rare pediatric disease
January 24, 2023

This article is posted on the UHN website.
Members of the team leading this innovative research project include, (L to R), Mary Vyas, President at PSC Partners Canada, Drs. Sonya MacParland, Senior Scientist at UHN's Ajmera Transplant Centre, Amanda Ricciuto, staff gastroenterologist at SickKids, and Gary Bader, affiliate scientist at the Princess Margaret Cancer Centre. (Photo: UHN)
A team led by UHN's Ajmera Transplant Centre, in partnership with the Donnelly Centre for Cellular and Biomolecular Research at the University of Toronto (U of T), The Hospital for Sick Children (SickKids), and PSC Partners Seeking a Cure, has received four-year funding of US$2 million from the Chan Zuckerberg Initiative to research the rare inflammatory pediatric disease, PSC.

What is unique about this project is that a patient partner organization, PSC Partners Seeking a Cure, is part of the research team as a co-principal investigator and as a funding recipient.

Patient partners will provide input throughout the study, including study design and priority setting, from the initiation of the grant writing to knowledge translation and dissemination of the results.

"Beyond incorporating the patient perspective in research design, our partners ensure that our work is grounded in the patient experience," says Co-Principal Investigator Dr. Sonya MacParland, Senior Scientist with the Ajmera Transplant Centre and associate professor in the Departments of Laboratory Medicine and Pathobiology and Immunology at U of T.

"Team-based science is the future," says team member and surgeon Dr. Ian McGilvray, who is Head of Liver and Pancreas Surgery at UHN's Sprott Department of Surgery, a surgeon and scientist at the Ajmera Transplant Centre, and associate professor in the Department of Surgery at U of T.

"All too often, we have been guilty of working alone or in silos. It's when we collaborate that we push science forward far more quickly."

"This grant marks a turning point not just for our rare disease organization, but for the broader rare disease research landscape in emphasizing the value patient partners can bring to research," says Co-Principal Investigator Dr. Ruth-Anne Pai, Director of Research Strategy at PSC Partners Seeking a Cure.

"At PSC Partners, we are thrilled to join this incredible research team working collaboratively towards a cure for PSC," she adds.

Such a study 'requires multi-centre coordination'

The patient partnership will also be supported by PSC Partners Canada.
"Receiving a diagnosis of PSC is devastating," says Mary Vyas, President at PSC Partners Canada, who is also a parent of a young adult living with PSC. "With no approved treatments, the best patients can hope for is a liver transplant and increased screening for early detection of potentially associated cancers.

"It is our mission to change this."

Primary sclerosing cholangitis (PSC) is a rare, immune-mediated liver disease, whereby, for reasons that are not fully understood, the immune system generates a dysregulated inflammatory response that damages the liver. It affects one in 500,000 children, and up to one in 100,000 adults every year. The condition causes inflammation in the bile ducts, leading to scarring of the liver. Most patients end up needing a liver transplant to survive.
Currently, very little is known about how PSC develops on a cellular level. However, most patients with PSC also have inflammatory bowel disease (IBD).

This research team is trying to understand the IBD link by finding shared cellular dysfunction in IBD and PSC. Finding these shared pathways could potentially lead to interventions that prevent the development or progression of PSC.

"PSC is such a rare condition that studying the cellular basis of its development requires multi-centre coordination," says Co-Principal Investigator Dr. Gary Bader, affiliate scientist at the Princess Margaret Cancer Centre and professor at U of T's Donnelly Centre for Cellular and Biomolecular Research.

"What is incredibly exciting and novel about this proposal is that it aims to study the liver and gut jointly," says Co-Principal Investigator Dr. Amanda Ricciuto, staff gastroenterologist at the Hospital for Sick Children and assistant professor in the Department of Pediatrics at U of T.

"This truly has the potential to unlock knowledge of key pathophysiological processes underlying the disease, and bring us closer to developing therapies."

This team and research are supported by UHN Foundation. Dr. Sonya MacParland holds a Tier 2 CRC in Liver Immunobiology. Once completed, the findings from this study will be made available with open access through the CZI's CELL by GENE platform and through the Human Cell Atlas Project, an international, open-access, collaborative effort to map all human cells.
Canadian PSCers in the News
From All Across the Country
We are introducing a new periodic feature in the Loon Duct to recap PSC & Canadian PSCers in the news! All across the country, PSC makes the headlines and often features patients working to spread awareness about disease, organ donation, or raising funds to support a great cause.
This story comes from Red Deer, Alberta and rdnewsNOW where Talbot Walton, who is the women’s volleyball head coach for Red Deer Polytechnic, described part of his PSC journey and the need for units of blood as part of the transplant process. On Feb 9, this local news outlet featured Talbot as part of the story as part of the Red Deer Polytechnic’s Student-Athletes Advisory Council working with Canadian Blood Services to promote donating blood.

Photo Credit: RDP Athletics

“Over the course of Walton’s medical procedures in the last eight years, he estimates he has used 12 units of blood and the need for more blood for people like himself or others in medical situations remains a priority.”
Although this second story is from last year on Feb 28, 2022, we still want to highlight it in case you missed it! This feature involves PEI resident Hara Kempton and the generosity of a friend willing to be a live liver donor and the story was featured on CBC’s The National. You can view the segment by following this link to the broadcast.



Hara Kempton (right) with friend and donor Heather Cry
Photo credit: CBC News, Hara Hempton

There was an accompanying written piece from CBC News about Hara’s story and the courage the comes from talking publicly about the need for organ donation. “Kempton called her campaign to find a donor "The Big Ask" and she said she never felt comfortable asking her friends directly so she just posted the information about the possibility of a live donor on social media and waited.”


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

Upcoming PSC Peer Group Zoom Rooms!

PSC Partners’ ZoomRoom meetings are a great way for Peer-to-Peer Connections!

Join others folks from the PSC community for these supportive, helpful, fun virtual events. Space is limited. Registration required. Link to register below. The sessions are not recorded, and are intended as safe open forums.

All at 6:00pm-7:00pm MT (8pm EST)
Unless otherwise indicated, events are listed in Mountain Time
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Tuesday, April 11, 2023

Are you a PSC patient, caregiver, or family member who feels underrepresented in the PSC community based on your race, ethnicity, gender, or other factors? This virtual support group is just for you!

 
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Wednesday, April 19, 2023
Caregivers, Spouses, and Parents of Patients with PSC and Post-Transplant 

Join this virtual peer-to-peer support group to chat with others who truly understand.

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

FOLLOW PSC PARTNERS CANADA ONLINE!
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.
Our mission is to drive research to identify treatments and a cure for primary sclerosing cholangitis (PSC), while providing education and support for those impacted by this rare disease.

PSC Partners Seeking a Cure Canada
A Canadian registered charity affiliated with
PSC Partners Seeking a Cure