Ground-breaking discoveries that provide hope of an alternative treatment option for haemophilia sufferers

Aug 11 2005

Haemophilia is a hereditary blood disease, primarily affecting males, where the blood fails to clot causing potentially life-threatening 'bleeds'. About one in 6000 Australian males is born with haemophilia in severe, moderate or mild form. People with haemophilia rely on intravenous infusion of recombinant Factor VIII clotting protein.

Professor Denisa Wagner and her Harvard colleagues have made ground-breaking discoveries that provide hope of an alternative treatment option for haemophilia sufferers.

Presenting this research, at the XXth Congress of the International Society on Thrombosis & Haemostasis in Sydney, Wagner said, "We have demonstrated that a protein called P-selectin is important for blood clotting and altering its levels in the bloodstream by infusion appears to have great therapeutic potential."

Infusion of P-selectin could provide an affordable and more effective means of achieving clotting to stop bleeding incidents in haemophiliacs. Because they carry it naturally in their bodies, patients are highly unlikely to make antibodies against P-selectin. P-selectin also has a longer half-life than clotting factors so treatment is likely to be less frequent.

"This promises to be a much easier and more effective approach for sufferers, particularly children," said Wagner.

Wagner went on to report, "Blood clotting is an intricately balanced process. Blood clots in the heart or brain can result in a heart attack or stroke. Our studies in mouse models have shown that inhibition of P-selectin reduces atherosclerosis (hardening of the arteries) and the work of our collaborators shows that the events leading to deep vein thrombosis are reduced. P-selectin inhibitors have also been shown to be anti-thrombotic in early human trials"

Measurement of P-selectin levels in the blood stream may provide a new diagnostic tool to identify people at risk of heart disease and stroke. Levels above baseline could alert clinicians to potentially fatal events. Several pharmaceutical companies are now searching for compounds that target P-selectin.

"This would represent a major breakthrough in diagnosing and treating cardiovascular disease," she said. "There is still a great deal to be learned about this protein. This research could contribute significantly to effective strategies to inhibit or enhance P-selectin depending on clinical need".