Novel Gene Therapy Potential Cure of Hemophilia A

Filed in archive Studies , Treatment on July 13, 2006

Affecting about one in 5,000 males, hemophilia A is a condition wherein one lacks the hereditary blood clotting protein, Factor VIII (FVIII). Treatment of this condition traditionally requires infusion of synthetic FVIII two to three times a week to control bleeding episodes.

Normally in an injury, platelets circulating in plasma - the liquid portion of the blood - stick to the site of the blood vessel wound, activating its surface and rapidly stopping bleeding. In hemophilia patients, infusion of FVIII replaces the missing clotting factor and enables normal cessation of bleeding.

But some 30% of these patients receiving infusion develop antibodies to FVIII thereby selectively inactivating its clotting properties and refusing its therapeutic role. In these patients, adults who have developed inhibitory antibodies, treatment can cost over $1 million annually if there is a major bleeding episode.

Well, there is good news to Hemophilia A sufferers as researchers from by Medical College of Wisconsin and BloodCenter of Wisconsin in Milwaukee have discovered a potential permanent genetic cure hemophilia A patients (including a subset who do not respond to conventional blood transfusions) in genetically altered hemophilia mice.

"We developed a Trojan horse approach cloaking FVIII in a platelet so that it is undetected by the antibodies and its clotting properties are preserved until the platelet sticks to a damaged blood vessel and releases its stored protein which now includes FVIII.," explains senior author Robert R. Montgomery, M.D., senior investigator at the BloodCenter of Wisconsin and professor of pediatric hematology at the Medical College.

Read more at EurekAlert.