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Why Do We Bleed, and How Can We Stop It?

Bleeding disorders have been around for thousands of years. But Dr. David Lillicrap, Canada Research Chair in Molecular Hemostasis, is providing new insights into the causes of these diseases, how they can be treated, and how to better manage the complications of treatment.

Lillicrap and his research team are using a combination of several molecular technologies to improve our understanding of what causes common inherited bleeding disorders, such as hemophilia and von Willebrand disease. They have already demonstrated important contrasting features in the genetic basis for these two diseases. Based on these findings, a National Reference Laboratory has been established to provide genetic testing for families with inherited bleeding disorders.

Currently, patients with an inherited deficiency of the clotting protein, factor VIII, are treated with frequent intravenous infusions of the missing factor. But 30 percent of treated hemophilia patients develop an immune response to the infused replacement protein. This response neutralizes the benefit of the infusions.

Lillicrap and his team have shed new light on the mechanisms responsible for this problem, and have demonstrated that intervening early to establish immune tolerance to factor VIII can significantly reduce the likelihood of this complication. They have already generated results with several innovative therapies that will form the basis of future treatment approaches. These novel therapies have shown that a long-term gene therapy cure of hemophilia is possible.