Investigators at Children's Research Institute, BloodCenter of Wisconsin's Blood Research Institute and the Medical College of Wisconsin have discovered a new way to help the blood clot by having the missing clotting factor packaged in the patient's own platelets.
In the October 2008 edition of Blood, investigators describe how a gene-modified bone marrow transplant can be used to initiate clotting in hemophilia. This type of approach may work in the 30 to 35 percent of hemophilia patients that have developed inhibitory antibodies against the missing clotting protein.
The bone marrow is removed from the patient and stem cells are treated with Factor VIII, a clotting factor, which is placed in the platelets. The marrow is given back to the patient, who then retains the essential clotting mechanisms to stop bleeding that otherwise would lead to complications.
For people suffering from hemophilia, this research means the potential relief of a constant, burdening disease. People who have hemophilia previously had to be treated every time they bled. Currently, they can receive treatments three times a week, but these are very costly and time consuming. The results from this study provide hope that people with hemophilia could potentially lead a disease-free life.
The scientific community once believed that hemophilia would be treated successfully by gene therapy. Research then showed that gene therapy typically resulted in the patient not retaining a substantial amount of clotting factor, which is integral in preventing serious bleeding.
This research was a collaborative effort that included investigators Qizen Shi, PhD, MD; David A. Wilcox, PhD; and Robert Montgomery, MD.