A research team at the Irish Centre for Vascular Biology, based at RCSI’s School of Pharmacy and Biomolecular Sciences, has identified how a new medicine can help people living with the bleeding disorder, von Willebrand disease, paving the way for more targeted treatments of the condition.
The most common inherited bleeding disorder in humans, von Willebrand disease, affects as many as one in 1,000 individuals. It is caused by a reduced quantity or function of von Willebrand factor in the blood.
This factor has an important role in blood to form clots, meaning that patients with von Willebrand disease experience abnormal bleeding episodes, such as heavy menstrual bleeding, nose bleeds, mouth bleeds and bleeding during surgery and childbirth.
At present, people with von Willebrand disease are treated by increasing von Willebrand factor in the bloodstream through drugs which encourage the release of von Willebrand factor that is stored in cells near blood vessels or by injecting von Willebrand factor into their veins.
New therapies and approaches
Through a series of lab experiments, the researchers showed how the new drug Rondaptivon pegol – also known as BT200 – helps to maintain levels of von Willebrand factor by slowing the removal of the factor from the blood by your immune system.
Specifically, it achieves this by making it more difficult for immune system cells called macrophages to bind to von Willebrand factor and remove it from the bloodstream.
This is a particularly promising development for patients who do not respond sufficiently to current treatment or who develop severe side-effects to current treatment.
It highlights how this clearance pathway can be a target for new therapies and may also lead to new treatment approaches for other blood diseases and disorders such as haemophilia A.
Scientific partnership
The research, which was supported by a Future (FFP) Award and the US National Institutes of Health, also involved scientists from BAND Therapeutics, who invented the BT200 drug.
The full paper, published in the prestigious journal Blood, is available here.
You can also learn more about RCSI's School of Pharmacy and Biomolecular Sciences here.
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