AEGiS-WSJ: Genentech Inc. Says Scientists Produce Rare Blood Protein

Important note: Information in this article was accurate in 1984. The state of the art may have changed since the publication date.

Genentech Inc. Says Scientists Produce Rare Blood Protein

Wall Street Journal - April 26, 1984
Marilyn Chase, Staff Reporter of The Wall Street Journal

SOUTH SAN FRANCISCO -- Genentech Inc. said its scientists have produced a synthetic form of rare blood protein used in the treatment of people who suffer uncontrolled bleeding.

The protein, called Factor VIII, induces clotting in normal people but is either missing or inactive in people who have hemophilia, a disease that strikes about one in 25,000 people. The genetic trait for the ailment is usually carried by mothers and passed on to their sons.

Currently, hemophiliacs take infusions of Factor VIII to stop bleeding from wounds or hemorrhaging into their joints, at a cost of $10,000 to $20,000 a year, Genentech said.

But currently available Factor VIII from such sources as Cutter Laboratories Inc. is extracted from purchased plasma, made from blood bought from poor people who are often ill. Thus it carries a risk of transmitting such blood-borne diseases as hepatitis and AIDS, acquired immune deficiency syndrome, a fatal collapse of the body's internal defenses against disease.

The Center for Disease Control in Atlanta has so far documented 30 cases of AIDS traceable to tainted Factor VIII, and recent publicity has prompted several recalls of the product.

Genentech said that several years of development, animal tests and human clinical trials must take place before its product is commercially available, however. Industry souces estimate the world-wide market for Factor VIII at about $250 million a year.

"We think it's wonderful news," said Alan Brownstein, executive director of the National Hemophelia Foundation in New York City. "This carries the potential for safer, more available, and perhaps cheaper Factor VIII. " Genentech, however, declined to give any information on its manufacturing or pricing plans for the substance.

Genentech's work involved isolating and cloning, or duplicating, the human gene that codes for normal Factor VIII production. Scientists then put the gene into mammalian cells and produced what they said was a pure and "biologically active" Factor VIII. When mixed with blood of hemophiliacs, this Factor VIII reduced their abnormally long clotting time to a normal rate of about 60 seconds from 100 seconds.

Genentech said the work involved 30 of its scientists working for more than a year, in cooperation with the Royal Free Hospital of London and Speywood Laboratories of Wrexham, England.
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