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Hemophiliac Treated in Britain with Substitute Blood Protein

October 11, 1988

LONDON (AP) _ A 25-year-old man has become the first hemophiliac in Britain to be treated with genetically engineered blood protein, a hospital said Tuesday.

If the blood-clotting protein proves safe, hemophiliacs may be able to avoid the risk of getting the deadly AIDS virus or hepatitis from donated plasma, scientists say.

The Royal Free Hospital’s Hemophilia Center and Hemostasis Unit said Victor Budgen of London was the first patient in Britain to be injected with the substitute protein, factor VIII.

About half a dozen hemophiliacs in the United States and West Germany have been treated in recent months with genetically produced factor VIII, he said.

Budgen ″responded excellently″ to his first injection last week and will be monitored for the next six months, said Dr. Peter Kernoff, director of the hemophilia center.

If the treatment is successful, it will be offered to other hemophiliacs, Kernoff said.

″There is an uncertainty associated with any new drug,″ Kernoff said in a telephone interview. ″I think in this case there is every reason for optimism because what is produced is virtually the same as natural factor VIII.″

Since about 1960, hemophiliacs have gotten factor VIII, which helps blood clot, from the plasma of unknown donors. But in the process they have risked contracting diseases contained in the plasma.

Factor VIII is missing in about 80 percent of the estimated 450,000 hemophiliacs around the world, according to a 1984 report by the World Federation of Hemophilia in Montreal.

The risk of AIDS contamination was reduced drastically three years ago by heat-treatment of all factor VIII supplies.

Kernoff forecast that it might be possible in the future to make ″designer factor VIII better than the body’s own system.″

Synthetic products might be developed ″that lasted longer in the blood″ than natural factor VIII, or could be taken by mouth instead of having to be injected, he said.

″Not only that but the knowledge gained in this project about the biology of factor VIII brings the possibility of a permanent cure for hemophilia, by gene therapy, very much closer,″ he said.

″The end objective is to cure hemophilia,″ he said. ″We’re expediting that process.″

The cost of treating hemophiliacs with the genetically produced protein has not yet been determined.

The process initially would be available mainly to hemophiliacs in the West, including some 5,000 in Britain, Kernoff said. About 85 percent of hemophiliacs worldwide, mostly in developing countries, ″are not treated at all,″ he said.

About 113 hemophilacs in Britain have developed full-blown acquired immune deficiency syndrome and an estimated 1,100 have been infected with the AIDS virus because of exposure to contaminated blood products, Kernoff said.

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