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Promising Therapies For Haemophilia & Heart Disease

August 11, 2005
Research Australia
Professor Denisa Wagner and her Harvard colleagues have made ground-breaking discoveries that provide hope of an alternative treatment option for haemophilia sufferers.

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 haemophiliain severe, moderate or mild form. People with haemophilia rely onintravenous infusion of recombinant Factor VIII clotting protein.

Professor Denisa Wagner and her Harvard colleagues have madeground-breaking discoveries that provide hope of an alternativetreatment option for haemophilia sufferers.

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

Infusion of P-selectin could provide an affordable and moreeffective means of achieving clotting to stop bleeding incidents inhaemophiliacs. 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 sotreatment 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 intricatelybalanced process. Blood clots in the heart or brain can result in aheart attack or stroke. Our studies in mouse models have shown thatinhibition of P-selectin reduces atherosclerosis (hardening of thearteries) and the work of our collaborators shows that the eventsleading to deep vein thrombosis are reduced. P-selectin inhibitors havealso been shown to be anti-thrombotic in early human trials"

Measurement of P-selectin levels in the blood stream mayprovide a new diagnostic tool to identify people at risk of heartdisease and stroke. Levels above baseline could alert clinicians topotentially fatal events. Several pharmaceutical companies are nowsearching for compounds that target P-selectin.

"This would represent a major breakthrough in diagnosing andtreating cardiovascular disease," she said. "There is still a greatdeal to be learned about this protein. This research could contributesignificantly to effective strategies to inhibit or enhance P-selectindepending on clinical need".

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