Treatment of common bleeding disorder may be boosted with new molecule

11 Jun 2020

Image: © Superrider/

RCSI researchers have tested a molecule that may help in the treatment of Von Willebrand disease, the most common genetic bleeding disorder.

Researchers from the Royal College of Surgeons in Ireland (RCSI) have made a breakthrough that may come as welcome news to people with a common bleeding disorder.

In a study published to the Journal of Thrombosis and Haemostasis, the researchers said they have found a way to promote blood clotting that could be used to help develop treatments for Von Willebrand disease, the most common genetic bleeding disorder.

Patients with Von Willebrand disease, which affects up to 1pc of the world’s population, 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.

Awareness of the disease is limited as the bleeding symptoms can appear mild, but the condition can result in severe bleeding from a serious injury or during surgery.

Now, using funding from the Science Foundation Ireland-Pfizer Biotherapeutics Innovation Award programme, the RCSI researchers and a team from Pfizer tested a molecule that extends the life of the clotting protein within the body’s circulation.

A long-acting therapy

This study showed that the modified molecule significantly increased the half-life by a factor of five compared with the unmodified protein in lab tests.

“The current treatments for Von Willebrand disease place a heavy burden on healthcare systems and for the patients themselves,” said the study’s lead author, Dr Jamie O’Sullivan.

“Long-acting therapies for the disease have the potential to reduce this burden. While more preclinical testing is needed, these are promising early results.”

In April, other RCSI researchers made headlines after they discovered that patients with severe cases of Covid-19 were experiencing abnormal blood clotting which, in some cases, led to deaths.

These micro-clots were found within the lungs and patients with higher levels of blood clotting had a significantly worse prognosis, requiring ICU admission. Authors of the study called for further research to investigate whether blood-thinning treatments may have a role in selected high-risk patients to reduce clotting and help them recover from Covid-19.

Colm Gorey was a senior journalist with Silicon Republic