Uniting Research Efforts to Create a Future Without PSC

PSC Partners International Collaborative Research Network

“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 launched the ICRNetwork at the Annual Conference in June 2022, and, together with the Reznek Family Center for PSC Research, is hosting the first in-person convening of PSC scientists in conjunction with the Annual Conference in September 2023! 

At PSC Partners, we continue to collaborate with PSC experts through the Research Grants program, Scientific/Medical Advisory Committee, our annual patient/caregiver conferences, the PSC Forum, attendance at national and international scientific meetings, and more.

Through the ICRNetwork, we will champion patient voices as we work with our partners around the world to:

• integrate international databases
• spark collaboration
• and facilitate patient-driven research projects and patient-focused drug development

Check out the sections below to preview our journey.

Downloadable Handouts

2022 PATIENT COMMUNICATION: DRAFT RESEARCH AGENDA

2022 DRAFT RESEARCH AGENDA

We hope these documents will serve as educational references and resources that you may come back to frequently as you consider your own hopes and needs for PSC research. Don’t forget that YOU are part of this Collective Network!

A Patient-Prioritized Research Agenda And Strategic Research Plan

The PSC Partners ICRNetwork will support the development of a Strategic Research Plan (SRP) with the goal of accelerating research towards treatments and a cure for PSC. The first step is to develop a patient-prioritized research agenda. PSC Partners has collected hundreds of research questions directly from PSC patients and caregivers through the 2021-2022 ROADMAP Initiative (Research Opportunities and Development Meetings to Accelerate Progress). In addition, the PSC Partners team has been meeting with expert PSC researchers to capture the most important research questions across various areas of PSC research. 

With hundreds of questions voiced by PSC patients, caregivers, researchers, and clinicians, PSC Partners will next look to the ICRNetwork to support the organization and prioritization of research questions into a draft research agenda. This draft research agenda was presented and discussed during the PSC Partners Annual Conference, June 2022.

PSC Partners is formulating a formal Strategic Research Plan (SRP) to guide the efforts of the PSC Partners ICRNetwork. With a SRP in place, PSC Partners will pursue specific patient-driven research projects over the next five years.

The PSC Partners Research Grants program will continue to support promising PSC research directions. With the implementation of the SRP, PSC Partners will expand efforts and capacity, and the ICRNetwork will drive forward key research projects prioritized by PSC patients. In our first round of prioritization, PSC Partners has focused on supporting drug development through the initiation of two multi-year projects: the PSC Symptom Assessment Project and WIND-PSC, the prospective synthetic cohort study. The PSC Partners Board of Directors decided to pursue both of these projects based on the needs voiced by PSC patients through the Our Voices survey and the Patient-Focused Drug Development (PFDD) Forum.

• PSC Partners WIND-PSC: A planned observational study of a prospective cohort of 2000 PSC patients from up to 15 different research centers in North America, UK and Europe.

• PSC Partners PSC Symptom Assessment Project, Measurement of how a person with PSC feels and functions: This project aims to ensure that PSC patient voices and symptom experience are heard loud and clear during clinical trials.

Pursuing The Most Important PSC Research Through the ICRNetwork

These research projects will meet key unmet needs for the PSC community, but our work together does not end here!

With the SRP in place, fundraising will be the next step to meet the budget of each research project. Fundraising isn’t the only way to get involved at this stage. Research projects cannot be completed without precious samples and health data donated by PSC patients. Your personal health data, blood, and tissue donations make research possible!

Together with the best experts to perform the research, our community funding and samples will propel research forward. Key findings from research projects will be shared openly with the full community of PSC patients, caregivers, researchers, clinicians, industry partners, and regulatory agencies. With further understanding of PSC, we’ll begin to ask new questions and pursue additional research, continuing the cycle. Ultimately, we hope that our collaborations will lead to patient-focused drug development and the identification of new treatments for PSC.

Patient-Focused Drug Development For PSC

While the aim of our SRP is to identify new treatments for PSC, these new treatments must first be rigorously studied in clinical trials. By initiating PSC Symptom Assessment Project and WIND-PSC, PSC Partners hopes we will be well-positioned to see meaningful improvement in PSC outcomes in future clinical trials.

Patients are at the core of all PSC Partners projects. PSC Partners strives to ensure a positive patient experience in clinical trials by providing the patient perspective to clinical trial design, as we recognize that no one knows the experience of living with PSC better than PSC patients. Through the ICRNetwork, patients and their caregivers will continue to help guide the development of clinical trials for PSC and respond to the Food and Drug Administration (FDA) requirement to pursue patient-focused drug development.

The PSC Partners Annual Conference

The PSC Partners Annual Conference looked a bit different in 2022 with the launch of the PSC Partners ICRNetwork. With this growing focus on patient-driven research, PSC Partners offered opportunities for patients and caregivers to meet and form connections with researchers and clinicians, who shared key research updates and welcomed input from PSC patients and caregivers. PSC Partners welcomed all conference attendees to join a session detailing the PSC Partners research agenda. We then provided the opportunity to join working groups to guide the development of the SRP.

With support from our conference collaborators, Dr. Josh Korzenik and Dr. Dan Pratt of Mass General Brigham, PSC Partners organized additional virtual sessions covering popular conference topics between July and December 2022. We welcomed expert researchers and clinicians to present and answer your questions through a dedicated Q&A general session. Some topics include PSC and IBD, symptom management, the basics of liver transplant, and an overview of ongoing clinical trials. 

CLICK HERE TO VISIT OUR YOUTUBE PAGE WITH RECORDINGS OF THESE PRE AND POST-CONFERENCE WEBINARS, AS WELL AS RECORDINGS OF 2022 CONFERENCE SESSIONS.

The 2023 Patient and Caregiver Conference will kick off with a report out from the working groups of the ICRNetwork who are working collaboratively to provide input on research projects that are aligned with the PSC Partners strategic research plan.

We Want to Hear What You Think!

Please email us at grants@pscpartners.org with feedback, questions, or suggestions.

You can also use the form below to send us your thoughts.