Hormone Offers Hope in Fight Against Infection

A genetically engineered hormone with the potential for revolutionizing the treatment of infections has passed its first human safety trial, UCLA and Harvard medical scientists said in a report published today.

In experiments conducted in Los Angeles and Boston, the hormone was given to 16 AIDS patients suffering from a wide variety of viral, bacterial and fungal infections that typically afflict people with acquired immune deficiency syndrome.

Two weeks of treatment with the hormone, known as GM-CSF, resulted in large increases in the levels of white blood cells that protect healthy individuals from acquiring those infections, according to Dr. Jerome E. Groopman of Harvard Medical School and Dr. David W. Golde and Dr. Ronald T. Mitsuyasu of UCLA.

Furthermore, the drug caused no life-threatening toxic effects, they said in an article in the New England Journal of Medicine.

AIDS patients are subject to many infections, such as pneumocystis pneumonia, because the AIDS virus causes a drastic drop in the level of the disease-fighting white blood cells known as neutrophils, monocytes and eosinophils.

Although it is not yet known whether the elevated level of white blood cells will actually protect patients, Golde expressed confidence in an interview that GM-CSF would have the desired effect because of what already is known about the hormone’s role as the regulator of the production and function of white blood cells.

Another trial to test the hormone’s efficacy is planned.

“Except for vaccines, doctors currently are unable to do anything to enhance a patient’s immune defenses,” Golde said. “But we expect to be able to regulate white cells (with GM-CSF) and to improve the ability to fight off diseases. GM-CSF can’t help but be useful.”

GM-CSF is a genetically engineered human blood cell growth hormone. Its potential uses in medicine, according to the researcher, go far beyond the treatment of AIDS.

GM-CSF and other recently discovered hormones that stimulate the growth of red blood cells are expected to reduce the need for blood transfusions during surgery and serve as a treatment of aplastic anemia, a condition in which the body no longer is able to make blood cells, and as a kind of antidote for toxic anti-cancer drugs that destroy blood cells.

Human GM-CSF, which stands for granulocyte-macrophage colony-stimulating factor, was discovered in 1966. It was first purified by Dr. Judy Gasson at UCLA in 1984. The cloning of the gene for the hormone in 1984 by scientists at Genetics Institute in Boston opened the way for the manufacture of large amounts of the compound for large-scale testing.