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
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.
Gene therapy
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 researchers succeeded in raising the concentration of coagulation factor VIII 1000 times.
Unfortunately, however, scientists ran into difficulties when testing the therapy on larger laboratory animals. After gene therapy, as is also the case with regular injections of coagulation factors, the immune system often recognizes these coagulation factors as ‘foreign’ and purges them, rendering the treatment ineffective. With the research project that has been selected to receive the award, the VIB research group will seek to identify which mechanisms are involved in the purging of this coagulation factor and how they might develop strategies to prevent this response.
Hemophilia Research Award
With its Hemophilia Research Awards Program, Bayer is supporting research on coagulation disorders in order to develop a better treatment for hemophilia patients. To be awarded such a research grant, the research project must be innovative, of high scientific caliber, and must offer added value to society. Thierry VandenDriessche’s research project was selected based on an international competition. The VIB researcher will receive 200,000 USD to carry out the project.
Promising research
With this research, the VIB scientists hope to explain the mechanisms that are responsible for causing gene therapy in hemophilia patients to ultimately fail. Knowledge of these mechanisms will play a key role in improving and developing therapeutic treatments.