Image: © kamiphotos/Stock.adobe.com
The discovery could pave the way for targeted treatments for von Willebrand disease, which is the most common inherited blood disorder.
The RCSI University of Medicine and Health Sciences has unveiled new research that shows a new medicine, Rondaptivon pegol, could help people living with the common bleeding disorder von Willebrand disease.
The main characteristics of the inherited illness, which are poor blood clotting and excessive bleeding, are caused by underperforming or low levels of von Willebrand factor, a protein that helps blood clot. The symptoms can be serious, for example, during surgery or childbirth, and sufferers often experience heavy blood flow from nose bleeds and menstruation. It is estimated that the disease could affect as many as 1 in every 100 people.
Dr Alain Chan Kwo Chion, a senior postdoctoral fellow at the RCSI School of Pharmacy and Biomolecular Sciences and the Irish Centre for Vascular Biology, explained that currently people with von Willebrand disease treat the condition by increasing von Willebrand factor in their bloodstream, through drug administration, encouraging the release of factor that is stored in cells near blood vessels or by injecting it into their veins.
RCSI’s research indicated that Rondaptivon pegol, also known as BT200, makes it harder for immune system cells to bind themselves to von Willebrand factor and remove it from the blood. The end result is that the factor will stay in the individual’s bloodstream for a longer amount of time. RCSI’s new discovery will clear the way for an increase in targeted treatments aimed at people living with the condition and for other disorders of the blood.
Prof James O’Donnell, from the RCSI School of Pharmacy and Biomolecular Sciences and director of the Irish Centre for Vascular Biology, noted the potential for this discovery to positively impact the lives of people living with blood conditions.
“Our data supports the concept that targeted inhibition of von Willebrand factor clearance pathways represents a novel therapeutic approach for von Willebrand disease and other blood diseases and disorders such as hemophilia A,” he said.
“In the case of von Willebrand disease, this novel therapeutic approach may significantly impact the lives of patients who do not respond sufficiently to current treatment or who develop severe side-effects to current treatment.”
The research was supported by a Future (FFP) Award and the US National Institutes of Health and involved scientists from BAND Therapeutics, who invented the BT200 drug.
Earlier this year, RCSI partnered with Serosep to find biomarkers that can predict the progression of inflammatory bowel disease to improve treatment options for patients.
Find out how emerging tech trends are transforming tomorrow with our new podcast, Future Human: The Series. Listen now on Spotify, on Apple or wherever you get your podcasts.
