Robert took the first cream-colored pill on April 14. Like the other 16 AIDS sufferers in Chicago's University of Illinois AZT drug study, the 27-year-old Chicagoan did not know if he was swallowing an experimental compound that might be the first effective treatment for his lethal disease or a placebo (a dummy pill). Neither did the nurses who gave the patients frequent blood and skin tests, nor the physicians who ran the "double-blind" human trial. Over the next five months, only half the volunteers were given AZT: The cruel nature of AIDS decreed that the surest proof of AZT's value would be a disproportionately high death rate in the control group taking the placebo. As the experiment continued at 12 hospitals, a common pattern emerged: Some patients gained strength, others wasted away as the disease ran its relentless, fatal course. In Chicago, one of the eight patients receiving placebos died during the test, and six others weakened from various opportunistic infections.

Robert, a former department manager in a housewares store, is one of the lucky ones. On Sept. 18, nurse Joanne Despotes called to tell him he'd been on the placebo but was getting an early birthday present—AZT. "I grabbed a cab and took off to the clinic to pick up the AZT right away," says Robert (who asked that his last name not be used). "I'd rather have been taking the drug all this time. But I understand the need for a placebo study. The AZT may buy me a few extra years. I may be around for a cure."

After five years and more than 14,000 deaths, the 11,000 surviving AIDS sufferers in the U.S. have been offered a glimmer of hope. In the course of the controversial AZT study, which began last February, 16 patients on the placebo died, compared with only one on the drug. Faced with those dramatic results, the Burroughs Wellcome drug company abruptly broke off the trial two weeks ago. On Sept. 19 the Department of Health and Human Services and Burroughs announced that AZT would now be offered free, not only to the placebo group but also to other eligible AIDS victims, mainly some of the estimated 6,000 who are suffering from Pneumocystis carinii pneumonia (POP). (The study did not focus on other categories of AIDS victims, and it is not known whether AZT will benefit them.)

Although placebo tests are an efficient way of evaluating a new drug's effectiveness, critics attacked the study for using patients as "human guinea pigs." In the Chicago group, a patient who required an ambulance to make his hospital visits wept openly when he learned he had been receiving the placebo, fearing his switch to AZT had come too late to save him. He died a few days later. "I feel badly for those who were not on the medication," says Michael Hyman, 39, a former vice-president of a central Illinois bank, who was put on AZT in the Chicago trial. "We are not dealing with athlete's foot. You are dealing with people like myself who have been diagnosed as terminally ill. Couldn't you test it in a study where everyone took the drug?"

A six-member medical review board, all independent of Burroughs Wellcome, was set up to halt the experiment if any unexpected harm or benefit showed up among patients. "We wanted to keep ourselves out of a Godlike role," says Dr. Dannie King, who headed the AZT study for Burroughs. Now, however, Burroughs must contend with the pleas of AIDS victims who still do not qualify for AZT. "I really feel sad that we can't wave a magic wand, but there are enormous medical questions to be answered," says King. "While I am very hopeful that this drug will show promise in other populations of patients, as a scientist I have to limit our conclusions to what we've done, not what we hope."

There was little hope at all for AIDS sufferers back in 1984 when Burroughs pulled azidothymidine—or AZT—off the shelf. Two decades earlier it had proved ineffective as an anticancer agent. But last year AZT produced encouraging results against the AIDS virus in lab tests, and most of the 19 patients in Phase I of a human trial showed signs of improvement. The stage was set for Phase II, a nationwide trial primarily limited to patients who had suffered their first attack of PCP within the previous four months.

The 282 volunteers had only a 50-50 chance of getting AZT, but word of the drug's potential spread quickly, and competition for it among AIDS victims was intense. Hundreds had to be turned away. In San Francisco, Rick Eastman's name was among 22 picked from a hat while he and a friend were in Mexico to find an illegal AIDS drug for an acquaintance. Eastman, 34, was given AZT—and ironically reports that he felt better six months ago, before he began suffering from the anemia that is the drug's most serious known side effect. Like others whose red-blood-cell count has dropped on AZT, Eastman receives blood transfusions about once a month to compensate.

During the trials, when a black market developed for AZT, with pills reportedly selling for $35 apiece in New York City, Burroughs kept the drug as secure as a narcotic. Some AIDS victims tried to bribe their way into the program, while others urged participants who thought they were getting AZT to share their medication. Jonathan Stewart, 28, who was admitted to the Miami trial three months after he nearly died from PCP, has seen two of his friends die from AIDS. A third is critically ill. "I've been watching his decline and seeing how sick he is," says the former Navy petty officer and divorced father of a 5-year-old daughter. "I feel like a lawyer went to the governor and at least got me a temporary stay." Stewart got steadily stronger; his weight went from a low of 130 lbs. on his 5'8" frame back to about 160. Stewart says he has had "safe sex" with a lover and, although he doesn't work out at a gym five times a week as he used to, he is doing "low-impact aerobics."

Andrew Meltzer, a 39-year-old conductor at the San Francisco Opera who has lost six friends to AIDS, was not as fortunate. He believed all along that he was on AZT, but his health continued to worsen during the study. He learned only when the trial ended that he was on the placebo. Meltzer is scheduled to enter the hospital soon to determine if a swollen lymph node is malignant. Three days after federal health officials made their announcement, he was told he would now be getting the drug. Meanwhile two of the original 12 patients on placebos in the San Francisco study have died.

Dr. King believes the human costs were worth the gain. "To those who may point at the 16 and argue that this study shouldn't have been done, I would say that it will have gotten this drug to more patients sooner, in a way that we can all be satisfied with ethically," he says. And the future now looks more promising. "This is the beginning," says King, "and it's a rather dramatic beginning."

  • Contributors:
  • Giovanna Breu,
  • Nancy Faber,
  • Margie Sellinger,
  • Laurel Tielis.