THE DUCT
A Monthly Newsletter for the PSC Community
March 2022
International PSC groups are already meeting to plan for PSC Awareness Day, which is October 29. Representatives of some of the groups are pictured above.
Together, we are creating a world where a PSC diagnosis comes with a cure!
Your voice is so important!
JOIN THE NEXT WEBINAR IN
THE ROADMAP INITIATIVE SERIES!
Gain a clear understanding of how patient participation in
natural history studies harnesses the power of the PSC patient experience
to drive research and drug development.

Natural History 201
Power of the Patient Experience:
Translating Natural History Data into Research Breakthroughs

Webinar Goals:

  • Join for the unveiling of the new PSC Partners natural history project, called the PSC Partners WIND (Worldwide Integration of Natural History Databases)
  • Discuss how patient data, like lab work and medical records, drives future research and drug development for PSC
  • Discuss why your patient voice and participation in natural history registries are so important

Following the presentation, attendees will participate in the breakout room discussions, including a room specifically for parents and caregivers of pediatric PSC patients.

The Breakout Rooms will be facilitated by:

  • Leina Alrabadi, MD, Stanford Children's Health
  • Dennis Black, MD, Le Bonheur Children’s Medical Center
  • Chris Bowlus, MD, UC Davis School of Medicine
  • Lisa Forman, MD, UC Health
  • Gideon Hirschfield, MChir, PhD, University of Toronto
  • Josh Korzenik, MD, Brigham and Women’s Hospital
  • Cynthia Levy, MD, University of Miami
  • Dan Pratt, MD, Massachusetts General Hospital
  • Kathy Siminovich, MD, University of Toronto
  • Kidist Yimam, MD, California Pacific Medical Center
Breakout rooms allow patients and caregivers to communicate directly with researchers, clinicians, and industry representatives. However, please know that, while sharing your voice is helpful and encouraged, it is not mandatory. It is perfectly fine for breakout room attendees to listen and learn. There is never any pressure to participate.

SEE ARTCLE BELOW FOR
MORE INFORMATION ABOUT WIND.
WATCH OUR ROADMAP INITIATIVE NATURAL HISTORY 101 RECORDING TO PREPARE FOR WEDNESDAY'S WEBINAR.
The following article comes from the most recent edition of the Loon Duct, a e-newsletter published by PSC Partners Canada. Click here to join the mailing list.
Announcing a New PSC Partners Initiative!
Worldwide Integration of Natural History Databases
What is WIND?
 
WIND, Worldwide Integration of Natural History Databases, is a PSC Partners-led and sponsored initiative that will make valuable patient data available to researchers. This will give them a better understanding of the lived PSC experience. 
 
The WIND database will serve as a central location where PSC natural history data is securely shared with PSC Partners and with the broader PSC research community. Medical centers from around the world will be invited to contribute clinician-reported natural history data. 
 
Data will be integrated from existing databases in different countries, amplifying their power by facilitating consistent, comprehensive, and monitored data collection. This will ultimately support and accelerate drug development for PSC.
 
Why is the natural history of a disease important?
 
Natural history refers to the usual course of a disease over time. When natural history is captured, patients are followed over time as their PSC progresses. With a patient’s consent, important data, such as treatments, complications, and more, can be stored securely in a natural history registry.
 
Natural history studies are crucial to PSC research, and will one day enable us to get the right treatment to the right PSC patients at the right time.

How is WIND different from a patient registry?
 
The main differences between a natural history registry (or database) and a patient registry are the types of data, and how the data is captured. The PSC Partners Patient Registry is a patient-reported registry. A natural history registry includes systematic and regular collection of past or current data from medical records, imaging data, hospitalization records, and lab test results. The systematic capture of these health data over time is key to enabling the completion of natural history studies.
 
For more on the basics of natural history studies, please consider watching (or re-watching) the recording of the ROADMAP Natural History 101.
 
What are some of the main objectives of WIND?
 
WIND aims to contribute to development of biomarkers which may be used to demonstrate effectiveness of an investigational drug for PSC. A biomarker is a characteristic of the body that can be measured. For example, blood tests or liver imaging may serve as biomarkers of treatment response. One reason clinical trials for PSC are challenging is because PSC currently lacks noninvasive biomarkers that are proven to correlate with disease progression. 
 
In rare disease drug development, natural history data may provide an untreated external control group (instead of a group receiving a placebo) for late-stage clinical trials. The natural history data must meet stringent regulatory collection and monitoring requirements, as well as patient confidentiality and data security; WIND is being designed with this use of the data as an objective.
 
The WIND initiative also plans to collect various measures of quality-of-life simultaneously with physician-reported clinical data. This will support the study over time of PSC symptoms and quality-of-life during PSC progression.
 
Who is involved in WIND? How can I join?
 
WIND is the inaugural project of the PSC Partners International Collaborative Research Network (ICRNetwork). The ICRNetwork is a place for anyone to volunteer and contribute to ongoing research efforts. The success of WIND relies on support from patients, caregivers, clinicians, and researchers. 
 
To support drug development, PSC Partners is working with great urgency to establish formal partnerships with academic researchers and institutions, and we will keep PSC Partners Registry participants and the community updated on which sites are ready to enroll patients into the WIND cohort. 

How does the PSC Partners Registry fit into this Initiative?

We value every PSC patient's voice. Regardless of whether you are able to join the WIND cohort, we encourage you to join the PSC Partners Patient Registry, through which you can give priceless input and support the WIND initiative and other PSC research studies. Through the Registry, PSC Partners may connect you to ongoing natural history registries, research studies, and clinical trials for PSC. 
Uniting Research Efforts to Create a Future Without PSC!
International Collaborative Research Network (ICRNetwork)
“Together in the fight, whatever it takes!” is a rallying cry for our community of PSC patients, caregivers, loved ones, and friends. For decades, PSC researchers and clinicians have continued the fight, investigating various treatments for PSC and working tirelessly to improve quality of life and outcomes for their patients.

PSC Partners has been inspired by the “Collaborative Network Model” defined by the Castleman Disease Collaborative Network (CDCN) in 2016. This model is described as a formal connection of key stakeholders with the same goal and core values: identifying treatments and a cure for rare diseases. In the spirit of this model, PSC Partners now invites researchers, clinicians, and industry partners to join our ranks as we unite our efforts through the PSC Partners ICRNetwork (International Collaborative Research Network). PSC Partners will officially launch the ICRNetwork at the Annual Conference, June 2-5, 2022.

Countdown to PSC Partners Annual Conference
June 2-5, 2022
This Conference Will Be Held Virtually
PSC Partners Conference By the Numbers:

75: Days until the conference
25: Suggested registration fee for patients and caregivers
0: Conference fee for folks who want to attend but can't cover the cost above
150: Registration fee for non-sponsor industry attendees
0: Registration fee for community and corporate sponsors
7: Scheduled pre-conference sessions to prepare for conference
11: Post-conference educational webinars
12: Number of educational presentations offered during the four-day event
8: Peer group, mentoring, networking and social opportunities available during the weekend

We are currently finalizing the agenda, which will be available soon. Until then, here's what can you expect from the virtual PSC Partners Conference:

  • Selective and engaging presentations on PSC and current research presented by international experts on PSC and PSC-related diseases, co-hosted with Brigham and Women’s and Mass General Hospital (Harvard University)
  • Opportunities to ask questions and have conversations with experts in small breakout sessions
  • Numerous chances to reconnect virtually with friends from the PSC community during fun social events, engaging peer-to-peer sessions, small group chats, and
  • Two days that are strictly for patients and caregivers (Thursday and Sunday)!

We need volunteers!

If you'd like to help with this year's conference, we need you! Visit the conference page on our website, and complete the volunteer form at the bottom of the page. Thank you!

Get more information about the 2022 PSC Partners Conference here. We are busy working on this exciting event, and will continually update this webpage. We'll inform the community as soon as registration is open.
Why Did You Come To PSC Partners Conference?
Each month, we highlight videos we think will be of interest to our community. At the 2019 PSC Partners Conference, several people let us know why they keep coming back. Click arrows on the graphics below to check out their answers.
REGISTER FOR AN UPCOMING ZOOMROOM
ZoomRooms
Are a Great Way To Make
Peer-to-Peer
Connections!
"Thank you for a wonderful support group for the devastating disorder of PSC.
May a cure be found sooner than later, whatever it takes!" - ZoomRoom Attendee
Have you joined a ZoomRoom, yet? These safe, open forums were started at the beginning of the pandemic to ensure our community members stayed connected and did not feel alone. They have been so successful that, even as the world is opening up, these virtual gatherings will continue. Attendees keep coming back, because of the comfort and support they feel when talking to others who truly understand what they're going through. If you haven't attended yet, please consider joining. If you know someone who might benefit from a ZoomRoom, please pass along this information.
Visit the EVENTS PAGE on our website for all scheduled ZoomRooms and other events of interest.

Help us keep our community informed by sending relevant events to PSC Partners! If you have an event you think should be added to the calendar, send information to contactus@pscpartners.org. Events will be screened for applicability to our community.
The #RAREis Scholarship is available to applicants who are:
·       Over the age of 17
·       Residents of the United States
·       Diagnosed by a physician as having any form of rare disease regardless of treatment status. Undiagnosed patients are also encouraged to apply. 
Must be planning to enroll full-time or part-time in undergraduate or graduate study at an accredited two- or four-year college, university, or vocational-technical/trade school for the Fall 2022 semester. There is no minimum amount of credit hours to be part-time. 
Welcome to Sandi's
Circle
To Conference or Not to Conference

"I wasn't going to go (to the PSC Partners Conference). Why? Well, I wasn't sure I had it in me, physically, emotionally, or otherwise. I was just plain, old spent. But, pal, boy am I glad I went, and here's why: You all rock. We rock. PSCers are amazingly extraordinary people and stunningly wonderful in so many ways.

At the conference, it doesn't matter if you're 13 or 33, 40 or 75, we're all the same, even in our differences, because we choose, on a level not even our subconscious recognizes as decision making, to recognize each other.

During sessions, we devour information and look to the experts to fill gaps in our knowledge or teach us something new. Out of sessions, we sing and we shop and we savor each moment. We're never in this thing all alone, because we have each other."
Folks who have been in the PSC Partners community remember Sandi Pearlman as a bright, positive light. Her insight, humor, and supportive presence were a real gift to those who knew her.
In this space, we share words of encouragement from Sandi and others.

If you have something to add to Sandi's Circle, send it to contactus@pscpartners.org.

Due to space limitations, not all submissions can be added, but we invite everyone to use this space to encourage folks in the PSC community.
Grab Some PSC Partners Branded Gear for Conference
The PSC Partners Conference may be virtual this year, but that doesn't mean you should skimp on your outfit. Even virtually, we can all represent! Take a look at the PSC Partners online shop, and pick up some PSC Partners gear before the June conference. We've got lots of styles to choose from to make sure you look and feel great!

The PSC Partners online shop is an awareness-raiser, not a fundraiser.
There are numerous ways to support PSC Partners. Check out our Ways to Give page for information. To make a donation, click here.

Thank you!
Our Mission: The mission of PSC Partners Seeking a Cure 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.
(303) 771-5227
6900 E. Belleview Ave., Ste 202,
Greenwood Village, CO 80111