VCU liver specialist partners with patient group to study rare disease’s impact
VCU receives grant to be one of three sites in U.S. for international study on primary sclerosing cholangitis.
October 22, 2024
Sayed “Obi” Aseem, M.D., Ph.D., is an assistant professor in the VCU School of Medicine’s Department of Internal Medicine and is part of the VCU Stravitz-Sanyal Institute for Liver Disease and Metabolic Health. (VCU Stravitz-Sanyal Institute for Liver Disease and Metabolic Health)
PSC Partners recently launched the Worldwide Initiative for New Drug Development in PSC (WIND-PSC), a groundbreaking project aimed at finding treatments for primary sclerosing cholangitis. VCU is one of three sites in the United States selected for the project and the only site in the Mid-Atlantic.
Sayed “Obi” Aseem, M.D., Ph.D., an assistant professor in the VCU School of Medicine’s Department of Internal Medicine, received a $445,526 grant to start VCU’s participation in the five-year project. “We are hoping to contribute more than 100 patients to the 2,000 patients (sought) for the study,” said Aseem, who is also part of the VCU Stravitz-Sanyal Institute for Liver Disease and Metabolic Health. Participants are already beginning to enroll for this study at VCU.
Primary sclerosing cholangitis, or PSC, inflames and scars the bile ducts, a network of small tubes that carry digestive fluid from the liver to the small intestine. The scarring can lead to cirrhosis, liver failure, transplant, or even death. An estimated 30,000 people have PSC in the U.S., according to PSC Partners.
“PSC is a rare liver disease, but with a significant disease burden, risk of malignancy and mortality. There are currently no treatments for PSC. So, in most patients, this is progressive to end-stage liver disease requiring a liver transplant or cancers of the liver,” Aseem said.
The National Institute of Diabetes and Digestive and Kidney Diseases reports that PSC is more common in people who:
- Are between the ages of 30 and 40
- Are male, affecting men twice as much
- Have inflammatory bowel disease
Symptoms can include abdominal pain, itchy skin, diarrhea, jaundice, fatigue, fever, or a bile duct infection. Research suggests that several factors may play a role, including genes, immune system problems, changes in the bacteria in the digestive tract, or bile duct injury caused by bile acids. The disease is typically diagnosed with blood and imaging tests.
WIND-PSC is creating a large, global database of information on PSC patients. This will include medical data as well as reports on symptoms and patients' quality of life. By gathering, analyzing, and sharing this data, researchers will have a much better understanding of how PSC progresses and what treatments may be most effective.
“The WIND-PSC initiative will drastically accelerate the process of treatment development by serving as a comparison cohort for other clinical trials of specific interventions. It will also provide valuable information for better disease biomarkers and how to improve the symptoms and quality of life for those afflicted by this condition,” Aseem said.
PSC Partners notes how close patient involvement is key to this initiative, so patients can shape the future of research on the disease and ensure that researchers understand what is most important to them.
“WIND-PSC is a unique project not only in its goals but in the partnership between the PSC clinical research and patient community,” Cynthia Levy, M.D., professor of medicine at the University of Miami and the project’s principal investigator, said in a statement. “By working together, we believe this project can accelerate drug development and finally have an effective treatment for this disease.”
The effort is bringing together patients, caregivers, researchers, doctors, and medical institutions in the U.S. and Canada. PSC Partners, along with PSC Partners Canada, has committed $4 million in funding to the project and plans to expand to 16 new sites in Europe with a total patient enrollment of 2,000. This funding will support research sites, help recruit patients, and cover the costs of managing and sharing the data.
Participants will be assessed annually with physical exams, lab tests, imaging, liver stiffness assessment, and markers for scarring, or fibrosis, of the liver. Clinical symptoms and events, medications, procedures, and patient-reported PSC symptoms will be gathered quarterly.
PSC Partners has previously supported Aseem’s research into this rare liver disease. Earlier this year, he received an $80,000 grant to continue studying a protein that may spur bile duct cells to produce more inflammatory substances and damage the duct and liver.
For more information on this initiative visit the PSC Partners website.
