The new treatment for hemophilia (lat. Haemophilia, also spelt as hemophilia) is being developed by the scientist from Western Washington University. Assistant Professor of Chemistry Clint Spiegel has been awarded a three-year, $390,000 grant from the National Institutes of Health to research potential treatments for hemophilia A, a hereditary genetic blood disorder that affects millions of people around the world. Patients with hemophilia A, the most common type of the disorder, have problems with their blood’s ability to clot or coagulate, which is the body’s natural defense to stop bleeding. Hemophilia is linked to a problem in the X chromosome and is far more likely to occur in males than females; approximately one in 5-10,000 males around the world has hemophilia A.
Spiegel’s research focuses on a protein involved in hemophilia A called the Factor VIII protein, which is deficient in patients with hemophilia A. Functional forms of Factor VIII are often used to treat hemophiliacs because of its ability to provide a short-term boost to the body’s ability to coagulate blood. Unfortunately, about 30 percent of those suffering from severe hemophilia A have antibodies in their systems which reject the Factor VIII protein, breaking it apart before it can assist the body in clotting and coagulation.
What Clint Spiegel and his team of graduate and undergraduate chemists hope to find over the next three years is a way to make the Factor VIII protein more stable and active and potentially more able to overcome the immune response of these hemophiliacs, making an improved version of the Factor VIII treatment a viable choice for potentially millions of sufferers of this disorder. In addition, they will look for ways to make Factor VIII less attractive to the antibodies that work to reject the protein.
Clint Spiegel said the research in the lab will involve crystallizing Factor VIII to look at its structures on a three-dimensional, atomic level and then using recombinant DNA techniques to make its protein structure tougher and more antibody-resistant. Clint Spiegel has had an interest in this subject area since he was a graduate student at the Fred Hutchinson Cancer Research Center; it’s something he is very familiar with and has worked on for years. There are also quite a few parallels between hemophilia research and heart disease research, and his father suffered from heart disease for more than 20 years, so he has got a personal interest in it as well.