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Researchers test new gene therapy to stop bleeding in hemophiliacs

PITTSBURGH (AP) -- Researchers at the University of Pittsburgh Medical Center are testing a new hemophilia therapy that could stop disease sufferers from bleeding.

Don Miller, 50, is the first patient to use the gene therapy designed by California-based Chiron Corp.For 43 years, Miller has been going to the hospital for treatment of hemophilia A, a disease that prevents his blood from clotting and causes severe bleeding in his joints and body cavities.

He uses a needle-administered treatment that stops bleeding after it starts, but the therapy could prompt his body to produce blood-clotting protein before he bleeds.

"No needle sticks," Miller said Thursday. "And I wouldn't be as afraid of hurting myself."

The gene therapy is injected into the veins of the hand and travels through the blood. The idea is for the friendly virus shell to carry the blood-clotting protein called factor VIII into the patient's body, where it will take up residence in cells.

"It's like a virus that's gutted of all its junk," said Dr. Margaret V. Ragni, professor of medicine at the University of Pittsburgh and director of the Hemophilia Treatment Center of Western Pennsylvania.

Hemophilia A is passed on genetically and affects only males. About one in every 10,000 men has the disorder, and pooling of blood in the joints often leads to crippling arthritis in later life when the enzymes in blood dissolve cartilage between the joints.

Miller is the first of what researchers hope will be several human test cases.

"With humans, we don't really know, and that's why we're very interested to be starting these trials," Dr. Deborah Hurst, who designs clinical trial plans and protocol for Chiron.