AMERICAN IDEAS, Page 18Guerrilla Drug TrialsThe Underground Test Of Compound QDesperate activists try to speed up the discovery of a curefor AIDSBy Dennis Wyss
Bob Barnett sits on an examination table in San Francisco while
an intravenous needle drips an experimental AIDS drug into his
veins. The drug, called Compound Q, is a purified protein extracted
from a cucumber-like Chinese plant and one of the latest promising
glimmers in the search for a cure for AIDS.
Across town, researchers at San Francisco General Hospital
Medical Center are conducting cautious, federally approved Phase
1 toxicity trials with minute dosages of GLQ223, as Compound Q is
officially known. But for Barnett, a 37-year-old former radio sales
manager, as for thousands of others afflicted with AIDS, precious
time is running out. Barnett wants to know if Compound Q works in
larger therapeutic doses. He wants to know now. "My options are
death and doing this," he says.
Barnett is one of 51 AIDS patients who, along with six doctors,
took part in underground trials of Compound Q this past spring and
summer. The clandestine study was organized by Project Inform, a
San Francisco-based group of activists who believe the Food and
Drug Administration's system for testing potentially life-saving
new drugs is unconscionably slow. On Sept. 19, Project Inform
director Martin Delaney revealed the preliminary results of the
underground trials to an intent crowd of some 500 predominantly
gay men in San Francisco. Although many of the trial's volunteers,
including Barnett, showed a marked decrease in activity of the
human immunodeficiency virus (HIV) that causes AIDS, Delaney said,
Compound Q could not be considered a cure. But the desperation of
the epidemic guarantees that underground drug trials will continue;
AIDS activists say at least two dozen such experiments are under
way across the U.S.
Hope flashed through the nation's AIDS community last April,
when researchers from the University of California at San Francisco
announced that, in test tubes at least, Compound Q could kill
HIV-infected cells while leaving healthy cells unaffected. The
substance quickly found its way into the U.S. and to desperate AIDS
patients, who administered the drug on their own. "Word was out,"
says Dr. Alan Levin, medical director of the Project Inform trials
in San Francisco. "People started getting it and injecting
themselves in their kitchens."
To Delaney, such haphazard self-medication posed its own
threats. "We said, `Instead of just passing it out to see what
happens, let's channel it into controlled clinical use,'" Delaney
recalls. He contacted James Corti, a Los Angeles-based activist and
importer of AIDS drugs who shipped 400 doses of Compound Q out of
China.
Delaney then asked a group of doctors to design a protocol, or
test model, based on an FDA trial for a similar drug called Ricin
Toxin. Delaney says several FDA and National Institutes of Health
officials in Washington were told of Project Inform's proposed
trial, which was planned for patients in San Francisco, Los Angeles
and New York City. "At no time did anyone tell us to stop," he
says. An FDA spokesman in Washington claims officials did not hear
about the clandestine trials until well after they began.
Without revealing the purpose, Project Inform asked Genelabs,
Inc., a California biotechnology firm that manufactures the drug
in the U.S., to test samples of Compound Q that Corti brought back
from China. They wanted to make sure it was identical to the
Compound Q used in the FDA-approved study. An attorney drew up
guidelines that would keep the trials within federal law. Each
patient made a videotaped statement, in the presence of an attorney
and a witness, that he was entering the trial of his own free will.
"What we wanted was a trial that was faster than the FDA, yet as
safe," says Dr. Larry Waites of San Francisco.
The trial's volunteers were all men who had failed to respond
to conventional AIDS therapy, including AZT, so far the only
FDA-approved drug for treating the AIDS virus. To obtain accurate
readings on Compound Q's effectiveness, volunteers were asked to
stop using any other approved or unapproved drugs.
The secret trials began on May 24 in San Francisco. For three
weeks, patients received infusions of Compound Q, some as high as
17 times the dosage given patients in the San Francisco General
Hospital toxicity trials. For the first 48 hours, the carefully
monitored volunteers suffered side effects of sore muscles, nausea,
fever and fatigue. The side effects eventually went away, and many
patients, including Bob Barnett, began to feel more energetic.
The clandestine study became public in late June after a San
Francisco volunteer suffocated on his vomit after coming out of a
coma ten days following his first dose of Compound Q. The FDA
launched an investigation into the underground trials, which
Project Inform suspended. Two other volunteers have since died, one
in San Francisco and one in New York. Levin says the death of one
of the San Francisco men was indirectly related to Compound Q,
while the cause of the New York man's death has yet to be
determined.
Some researchers raise serious doubts about the methodology of
guerrilla drug tests. Project Inform is strongly criticized for
bypassing an initial phase to establish Compound Q's safety before
proceeding to larger, therapeutic dosages and for not having the
trials reviewed by an external monitoring group. Says Jere Goyan,
dean of the University of California at San Francisco School of
Pharmacy and a former FDA commissioner: "If you get people taking
these drugs willy-nilly around the country, you'll lose valuable
information, and it will be at the expense of future patients."
To Delaney, such reasoning is flawed because it suggests that
some victims who might be helped by experimental drugs may die
while the traditional methods of testing drugs work their slow and
cumbersome way. Pressure from AIDS activists has resulted in the
FDA's allowing wider use of such experimental AIDS drugs as
r-erythropoietin, which is used to treat AIDS-related anemia,
before studies have been completed. Compound Q faces much more
rigorous testing despite the hint of promise. "It's not a one-shot
cure," Delaney warned the packed community meeting. But Bob
Barnett, a true believer in his right to receive another dose of
Compound Q, leaped to his feet with the rest of the crowd to give
