Improving treatment for Von Willebrand Disease

Scientists have found a potential new way to promote blood clotting that could be used to help develop treatments for Von Willebrand Disease, the most common genetic bleeding disorder.

Led by researchers at RCSI University of Medicine and Health Sciences, the study is published in the Journal of Thrombosis and Haemostasis.

Patients with Von Willebrand Disease have a deficiency or dysfunction in a protein that plays a key role in clotting blood. The current treatment for the disease is to frequently inject patients with a drug that promotes the production of this protein or with concentrations of the protein itself.

Funded by the SFI-Pfizer Biotherapeutics Innovation Award Programme, researchers from RCSI and Pfizer tested a modified molecule that extends the life of the clotting protein within the body’s circulation. Their study found that administering this modified molecule significantly increased the half-life fivefold compared to the unmodified protein in laboratory models.
While more preclinical testing is need, these are promising early results.

Von Willebrand Disease is an inherited bleeding disorder that affects up to 1% of the world’s population. Awareness of the disease is limited since patients’ bleeding symptoms can be mild. However, they can suffer from severe bleeding when there is a serious injury or need for surgery. Current treatments place a heavy burden on healthcare systems and patients. Long-acting therapies for the disease have the potential to reduce this burden.

RCSI University of Medicine and Health Sciences is ranked number one globally for Good Health and Well-being in the Times Higher Education (THE) University Impact Rankings 2020. RCSI is among the top 250 of universities worldwide in the THE World University Rankings (2021) and its research is ranked first in Ireland for citations. It is an international not-for-profit university, with its headquarters in Dublin, focused on education and research to drive improvements in human health worldwide. RCSI has been awarded Athena Swan Bronze accreditation for positive gender practice in higher education.

From time to time, RCSI will share research and education news of legitimate interest to you as a valued peer and colleague. You are included in this audience based on one of the following: opt-in, recent RCSI education/research event participation, nomination by an education/research collaborator or peer at RCSI. If you wish to stop receiving this news, please click "unsubscribe" below. RCSI's Privacy Policy is available here.