Important note: Information in this article was accurate in 1984. The state of the art may have changed since the publication date.
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Wall Street Journal - November 1, 1984
Marilyn Chase, Staff Reporter of The Wall Street Journal
AIDS is a viral disease that ravages the body's immune system, making way for so-called "opportunistic infections" that usually result in death. These secondary infections include rare varieties of pneumonia and cancer.
In a small human trial involving six AIDS patients suffering from pneumocystis carinii pneumonia, all improved after treatment with a drug known as DFMO, difluoromethylornithine. DFMO is an anti-parasitic drug manufactured in Strasbourg, France, by the Merrell Dow unit of Dow Chemical Corp. and used mainly in the treatment of African sleeping sickness.
The study, soon to be published in the November issue of the Western Journal of Medicine, was conducted by Jeffrey Golden and Daniel Santi of the University of California at San Francisco, and by Albert Sjoerdsma of Merrell Dow Research Institute of Cincinnati.
After six to eight weeks of DFMO therapy, the researchers reported patients' lungs cleared and their breathing eased. However, five of the six patients in the study subsequently died of other opportunistic infections stemming from AIDS. One of the patients is still living.
The DFMO was administered only to patients who didn't respond or couldn't tolerate two more-conventional drugs: pentamidine, which can cause kidney failure over time, and TMP-SMX, a combination of trimethoprim and sulfa that can induce a drop in white blood cells or an allergic response.
By mid-October, more than 6,400 cases of AIDS had been reported, including 3,000 deaths.
In patients who suffer the AIDS-related pneumonia, "basically what happens is that they can't get enough oxygen into their blood," Dr. Santi, a biochemist and pharmacologist, said. With treatment, he said, their blood's oxygen levels increased.
"We hope this drug will be effective against the pneumonia, but it will not cure AIDS itself," Dr. Golden, a lung specialist, said. Even so, results were dramatic in temporarily holding the pneumonia at bay. "Our first patient was heading for the intensive care unit, ready to die. With treatment, he turned the corner and lived for a year without symptoms," he said. A number of patients were able to return to work.
Both researchers cautioned that DFMO is an experimental drug, and that more human study is needed. Dr. Golden said he currently hopes to test the drug in another six to eight patients with an early form of the pneumonia.
Side effects of DFMO can include stomach disturbances, hearing loss or a drop in blood platelets, Dr. Santi said.
DFMO belongs to a family of drugs drawing increased attention in research circles. Such drugs, until recently poorly understood, are now known to work by means of a mechanism called "suicide enzymes" or the "Trojan-horse alternative."
The strategy is to chemically ambush an enzyme critical to the disease process, and is thought to hold promise in the treatment of epilepsy and cancer, as well as sleeping sickness and AIDS-related pneumonia, according to Paul Ortiz de Montellano, a University of California chemistry professor.
Along with Merrell Dow, companies known to be pursuing "suicide enzymes" include Merck & Co. and a Canadian unit of Syntex Corp., Mr. Ortiz said.
Dr. Samuel Broder, chief of clinical oncology at the National Cancer Institute in Bethesda, Md., said he too is conducting DFMO tests in about 10 AIDS patients with pneumonia, but "it's really too early to talk about results."
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