Drugs From The Underground

By Dick Thompson

As the AIDS epidemic progresses, the disease is doing more than snuffing out individual lives. The virus is attacking the all-important system the U.S. uses to test new drugs. Under pressure from AIDS activists, the Food and Drug Administration announced last week that it would allow wider use of two experimental drugs before rigorous clinical trials have conclusively established the value of these medications. AIDS patients hailed the decision, but it set precedents that could weaken the scientific safeguards that have long protected the desperately ill from quack remedies.

Even as the FDA was easing its rules, AIDS sufferers were still searching for a cure on the black market for unapproved drugs. It was revealed last week that an underground network of doctors in four cities has been conducting a clandestine trial of a drug known as Compound Q. In test tubes, it can destroy cells infected with the AIDS virus, but it has not yet been proved to be safe and effective in humans. In the unofficial trial, 42 patients have received Compound Q, which is derived from a Chinese cucumber-like plant. Among those taking the drug is Robert Pitman, 48, of San Francisco. Says he: "I was prepared to get it however I could. I was desperate enough that I would have injected it in my own living room."

The secret study, organized by a San Francisco-based group of AIDS activists called Project Inform, came to light after one of the patients died. He went into a coma, later awoke but then choked while vomiting -- ten days after his first Compound-Q treatment. The FDA has launched an investigation of the study.

The Compound-Q affair has heightened concern about the widespread use of unproven drugs. "There is always a tension between treatment of a patient and the need for solid drug testing," says Dr. Frank Young, the FDA commissioner. But AIDS has increased that tension. Those with the disease have protested for years that the FDA's traditional methods of testing an experimental drug's safety and effectiveness were too slow. "People have lost faith in the system," says Richard Dunne, executive director of Manhattan's Gay Men's Health Crisis.

Beginning in 1984, the FDA permitted the Syntex pharmaceutical firm to give doctors free ganciclovir, a drug used to treat eye infections that frequently blind AIDS patients, under a special program that allows "compassionate use" of unproven drugs. Doctors who have dispensed the drug are convinced that it works, but all the conventional controlled studies have not been done. Nonetheless, the FDA last week approved ganciclovir for full marketing and sales. The agency also gave the go-ahead for wider distribution of another unproven drug, erythropoietin, which is used in cases of AIDS-associated anemia.

Although FDA officials dispute the notion, some experts are concerned that the use of unproven medications may be getting out of control. So many AIDS patients are taking a pharmacological stew of approved and experimental drugs and potions that it is difficult to gauge the effectiveness of any single drug. Underground studies of experimental drugs, like the Compound-Q effort, confuse an already complex situation and frustrate scientists. "They're violating all the standards of safe testing of new compounds," says Dr. Paul Volberding, an AIDS specialist at the University of California at San Francisco. The haphazard use of experimental drugs may help some AIDS patients in the short run, but it will slow down the quest to discover the best ways to treat the many people who will contract the disease in the future.

With reporting by Jerome Cramer/Washington and Dennis Wyss/San Francisco