Leuven, Belgium − On 10 July, Thierry VandenDriessche, a researcher at VIB and the Katholieke Universiteit Leuven, is to receive the Hemophilia Research Award for his research on hemophilia − a hereditary disorder affecting coagulation of the blood. The research grant, for the amount of 200,000 USD, is being presented by Bayer at a meeting of the International Society of Thrombosis & Hemostasis in Geneva. With this prize, Bayer is lending its support to research on hemophilia. Thierry VandenDriessche and his colleague Marinee Chuah are investigating the possibilities of improved treatments for hemophilia, including gene therapy.
Hemophilia is a hereditary disorder in which certain coagulation factors in the blood are missing. This causes frequent bleeding in the muscles and joints, with tissue damage as a result. If the disorder is left untreated, the blood will no longer be able to coagulate, which can indirectly even lead to death. In 85% of the patients, there is a shortage of coagulation factor VIII. This condition is called hemophilia A. The rest of the patients most often have a shortage of coagulation factor IX (hemophilia B). The disorder has a frequency of 1 in 10,000. In the 20th century, hemophilia was one of the most well-known and extensively studied hereditary disorders, as it afflicted several royal families. Alexis, the only son of Russian czar Nicolas II, was perhaps the most famous hemophilia patient.
Regular injection of the right coagulation factor is usually the only possible treatment for hemophilia patients. Gene therapy might well be an alternative solution. In this approach to curing the disorder, a malfunctioning gene (part of the DNA) is replaced by a properly functioning gene. VIB researchers Thierry VandenDriessche and Marinee Chuah are the first to have achieved a sustained gene therapy cure for hemophilia A in mice. In the mice with hemophilia A, the r
Contact: Evy Vierstraete
VIB, Flanders Interuniversity Institute of Biotechnology