Stories about: von Willebrand disease

“Shapeshifter” that regulates blood clotting is visually captured for the first time

GIF of VWF, which regulates blood clotting, elongating and relaxing on loop
A single molecule of von Willebrand factor is visually captured, as it elongates and relaxes in response to blood flow conditions, for the very first time. Credit: Springer/Wong labs (Boston Children’s Hospital and Harvard Medical School)

We are normally born with a highly sophisticated array of molecules that act as “sentries,” constantly scanning our bodies for injuries such as cuts and bruises. One such molecular sentry, known as von Willebrand factor (VWF), plays a critical role in our body’s ability to stop bleeding.

To prevent hemorrhage or life-threatening blood clots, VWF must strike a delicate balance between clotting too little or too much. Researchers have long suspected that the mechanical forces and shear stress of blood flow could be closely-related to VWF’s function.

“In some ways, like in the movie Star Wars, VWF may be considered a Jedi knight in our body that can use ‘the force’ to guard the bloodstream,” says Timothy Springer, PhD, of Boston Children’s Hospital and Harvard Medical School (HMS).

It has not been possible to witness exactly how VWF senses and harnesses these mechanical forces — until now.

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A long wait, but worth it: Vonvendi’s Boston origins

blood vessel von willebrand factor Vonvendi

It’s no secret that it can take years, even decades, for a biological or medical discovery to move from the laboratory to the bedside. The Pharmaceutical Researchers and Manufacturers of America estimates that it takes on average at least 10 years (and $2.6 billion) to develop a new medicine from start to market.

But some wait times stand out. Take the story of Vonvendi, a recombinant form of von Willebrand factor (vWF), a clotting protein implicated in von Willebrand disease, a bleeding disorder.

Stuart Orkin, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and his then-research fellow David Ginsburg, MD, first cloned the gene for vWF in 1985. Pharmaceutical company Baxalta, which has been developing Vonvendi based on Orkin and Ginsburg’s original discovery, won FDA approval for the drug at the end of 2015.

But what makes this story unique isn’t the 30-year lag. Rather, it’s that — for reasons that aren’t entirely clear — the patent Orkin and Ginsburg filed for vWF in 1985 hung in limbo until 2013. But today, both men agree that while the wait was long, seeing their discovery emerge as a treatment is thrilling to no end.

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