Synthetic Clotting Agent Safer for Hemophiliacs

An Orange County man admits he is a guinea pig and doesn’t know the risks but the hemophiliac said Saturday he hopes to help find a cure for the disease that afflicts 20,000 Americans.

“I’m nervous as hell,” said 30-year-old computer software designer Chris Pitkin from Yorba Linda, who is taking a genetically engineered blood-clotting agent designed to reduce the risk of contracting AIDS.

“You don’t really know what you’re getting into,” Pitkin said. “The risks of a new technology are in a lot of ways unknown.”

For the past year Pitkin, and a select group of hemophiliacs around the country, have taken a synthetic version of factor VIII, the clotting agent missing from the blood of 80% of individuals with hemophilia.

Synthetic factor VIII is in the final stages of testing and may be commercially available by the end of 1990, following approval by the Food and Drug Administration.

Factor VIII was developed mainly by two pharmaceutical companies, Genetics Institute of Cambridge, Mass., and Genentech, headquartered in San Francisco.

Pitkin said hemophiliacs in the clinical trials must record each factor VIII infusion and transmit the data to researchers using a computer system he developed though his company, Caremark Inc.

Hemophilia is a hereditary blood-clotting disorder mainly affecting males. The National Hemophilia Foundation said hemophilia A, or factor VIII deficiency, has been treated effectively since the late 1960s with a factor VIII concentrate derived from human blood plasma.

The foundation reports 20,000 people nationwide have the disease, which affects one male baby in each 4,500 live male births.

Alan P. Brownstein, NHF executive director, said the synthetic factor VIII is safer than clotting agents derived from blood plasma because it removes the possibility the product could be tainted by a virus, such as human immunodeficiency virus, which causes AIDS.

“As long as people with hemophilia are dependent on products derived from human source material for their treatment, there will always be the potential danger of viral contamination,” Brownstein said.

Pitkin said the spread of the AIDS virus prompted researchers to speed development of the synthetic product to eliminate the possibility of hemophiliacs becoming infected through tainted infusions.

Pitkin said hemophiliacs lead relatively normal lives but must monitor themselves for internal and external bleeding. Synthetic factor VIII, as with blood-based clotting agents, is taken intravenously as needed.