DENVER & TORONTO--(BUSINESS WIRE)--PSC Partners and PSC Partners Canada, both patient-led non-profit organizations supporting education and research in primary sclerosing cholangitis (PSC), announced today the enrollment of the first patient into the WIND-PSC study. PSC is a rare life-threatening chronic liver disease which can lead to cirrhosis, liver failure, transplant, or death. Additionally, patients can experience significant symptom burden and compromised quality of life as their disease progresses. Currently, there are no regulatory-approved therapies for PSC and the development of new treatments remains challenging due to the rare nature of the disease. WIND-PSC has been developed to generate key data to remove some of these barriers and accelerate the development of new treatments.
WIND-PSC will recruit approximately 2,000 patients and gather clinical, biomarker, and patient-reported data about the progression of PSC and its associated symptoms. To support the design and implementation of the study, the PSC Partners team led by Stephen Rossi, PharmD, Chief Scientific Officer, and Mary Vyas, President, PSC Partners Canada, brought together a Steering Committee of global PSC experts comprised of Christopher Bowlus, MD, University of California - Davis, Bettina Hansen, PhD, Erasmus University, Gideon Hirschfield, MD University of Toronto, Cynthia Levy, MD, University of Miami, and Christoph Schramm, MD, Universitätsklinikum Hamburg-Eppendorf. Arbor Research Collaborative for Health is supporting this effort by serving as the Data Coordinating Center and providing other key clinical trial services.
“WIND-PSC is a unique project not only in its goals but in the partnership between the PSC clinical research and patient community,” says Cynthia Levy MD, Professor of Medicine at University of Miami and Principal Investigator for WIND-PSC. “By working together, we believe this project can accelerate drug development and finally have an effective treatment for this disease.”
The collected data will provide a rich and novel source of information to be shared widely with the PSC research community. The primary aims of WIND-PSC are:
- Develop a prospective real-world data cohort to support the design of PSC clinical trials and support regulatory filings for new treatments
- Evaluate key biomarker data, including the collection of bio-samples, to establish acceptable endpoints for use in clinical trials
- Assess patient-reported symptoms and quality of life, and other direct patient experiences with standardized tools to determine changes with disease progression
Patient participants will be enrolled at up to 20 sites in North America and Europe and will be followed for up to five years. All participants will be assessed annually with physical exams, lab tests, imaging, liver stiffness assessment, and fibrosis biomarkers. Clinical symptoms and events, medications, procedures, and patient-reported PSC symptoms will be assessed quarterly.
“The movement towards patient-led research is a momentous step for PSC Partners but a challenge we are willing to take head-on,” says Stephen Rossi, PharmD. “There are many stakeholders in addition to the PSC patient and research community who are key to the success of WIND-PSC, including the regulators, pharmaceutical companies, and other patient organizations. We have been actively engaging and collaborating with all of them as we move into this important first step of launching the project.”
“This project represents the culmination of the many years our organization has supported novel research to find a cure for PSC,” says Ricky Safer, Founder and CEO of PSC Partners and a patient living with PSC. “The community spoke up to us clearly regarding their priorities for research and we have taken their voice to heart with WIND-PSC.”
About PSC:
PSC is a rare chronic cholestatic liver disease characterized by diffuse inflammation and fibrosis of the bile ducts (Assis 2023). An estimated 30,000 people have PSC in the US, with one in 10,000 persons having this disease globally. Disease progression may result in cholestasis, cirrhosis, liver failure, as well as an increased risk of hepatocellular cancer and cholangiocarcinoma (Sohal 2024). Liver transplantation remains the only clinically proven treatment, though up to 20% of patients have recurrent disease (Leung 2021). There are no therapies approved by regulatory authorities for the treatment of PSC or related clinical symptoms and a significant unmet medical need still exists for new treatments.
About PSC Partners
Founded in 2005, PSC Partners Seeking a Cure is a 501(c)(3) nonprofit organization. The various programs facilitate and support patients, caregivers, families, and friends, educate patients and the medical community about PSC and support groundbreaking research in search of treatments and a cure. PSC Partners, headquartered in the United States, extends its impact globally through initiatives such as an international patient registry, international research grants, an affiliated Canadian registered charity, collaborations with other PSC organizations, and support of the worldwide PSC community. More information: pscpartners.org
Assis DN, Bowlus CL. Clin Gastroenterol Hepatol. 2023 Jul;21(8):2065-2075.
Leung KK, Deeb M, Fischer SE, Gulamhusein A. Semin Liver Dis. 2021 Aug;41(3):409-420.
Sohal A, Kayani S, Kowdley KV. Clin Liver Dis. 2024 Feb;28(1):129-141.
