Monday, Jan 12, 1981

FDA-Approved Clinical Tests on Humans Begin Today With Human Growth Hormone Made by Recombinant DNA

South San Francisco, Calif. -- January 12, 1981 --

Genentech, Inc., announced today approval by the Food and Drug Administration to proceed with human clinical trials of human growth hormone (hGH) made by recombinant DNA technology.

The Phase I study, which began today, is being conducted at the Department of Pediatrics of the Stanford University School of Medicine, Palo Alto, CA.

In February clinical trials of Genentech's human growth hormone will begin in the United Kingdom, at London's Great Ormond Street Hospital for Sick Children.

"The initial hGH trial at Stanford, in which biosynthetic hGH will be tested for safety in normal, healthy adult volunteers, is expected to last 20 days," Robert Swanson, President of Genentech, explained. "After that," he continued, "studies of effectiveness as well as safety will be undertaken in several academic medical centers throughout the country on patients 4 to 13 years old who are suffering from hypopituitary dwarfism (short stature caused by deficiency of growth hormone). Those studies will be conducted for a period of one year."

According to the Human Growth Foundation, an estimated half million children in the United States currently suffer from various growth problems. In the past, the only source of hGH has various growth problems. In the past, the only source of hGH has been human pituitary glands derived from cadavers at autopsy.

It takes 50 cadavers to provide enough hGH to treat a single child for a year. The resulting limited supply has not only restricted treatment of hypopituitary dwarfism, but hindered investigations of hGH effectiveness in other growth disorders was well.

Unlike insulin and some other hormones, animal growth hormone is not effective in humans.

The greatest price of the Genentech development is that clinicians may now enjoy unlimited supplies of hGH for treatment of hypopituitary dwarfism as well as for assessment of beneficial effects in other forms of growth disorders.

In the December 1980 issue of Research Resources Report, published by the government's National Institutes of Health, Henry Miller, M.D., medical officer of the Food and Drug Administration's Division of Metabolism and Endocrine Drug Products in the Bureau of Drugs, was quoted as saying about hGH produced by recombinant DNA techniques:

"This will provide potentially limitless supplies of hGH for treating both the short stature of pituitary origin and for clinical studies for other indications."

It is known that hGH can stimulate growth in many children whose short stature is not directly attributable to hypopituitarism. These children can have their height increased by up to four inches a year with the administration of hGH. It is estimated that several hundred thousand U.S. children could thus potentially benefit from hGh therapy.

Among the other potential applications of hGH are these:

  • The possibility that hGH may help to hasten the repair of wounds, non-healing fractures and torn cartilage.

  • The possible treatment of short stature associated with Turner's Syndrome, which affects one in 2,500 females born in the United States each year.
  • The possible treatment of juvenile rheumatoid arthritis and osteoarthrosis.
  • The possible treatment of serious burns, stress ulcers, hypercholesterolemia, and osteoporosis.

Promising studies have been done in most of these areas, but the amount of available hGH has been so limited that conclusive results could not be obtained. The December 1980 issue of Research Resources Reporter quoted Dr. Daniel Rudman, Professor of Medicine at Emory University, as saying:

"We haven't seen any side effects in our experience with ... children treated with pituitary-derived hGH. I'd say the general view is that after 20 years of experience in more than 100 institutions all over the world, this treatment seems remarkable free of side effects."

Human growth hormone is the second product of the Genentech recombinant DNA research to reach the state of human clinical trials. In November Eli Lilly and Company began human clinical trials of biosynthetic insulin produced under an agreement with Genentech.

For both hormones, recombinant DNA techniques hold the promise of unlimited supplies.

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