A Gene Cure For Hemophilia?

For 43 years, Don 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 lives carefully. He loathes his constant companion -- the needle-administered treatment that stops bleeding after it starts.

Researchers at the University of Pittsburgh Medical Center are testing a new therapy on Miller that could prompt his body to produce the blood-clotting protein it lacks before he bleeds.

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

Miller, 50, is the first patient to use the gene therapy designed by California-based Chiron Corp. as part of a clinical test. 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 body, where it will take up residence in cells.

"It's like a virus that's gutted of all its junk," said Dr. Margaret 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.

The treatment has been tested on mice, rabbits and dogs. Some animals showed results just two weeks after receiving the treatment, and dogs showed continued production of the protein for a year after it was administered.

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.

Written By Paula Story