SAN FRANCISCO, JUNE 22 -- Preliminary results from a four-month trial of the controversial and experimental AIDS drug Compound Q, released at the International Conference on AIDS here today, showed that 38 of 46 patients experienced a stabilization or measurable improvement in their condition.
The study -- the first major test of an AIDS drug to be initiated outside the normal federally approved system -- raised hope among AIDS activists and protests among many AIDS researchers. The study suffered from what many consider a near-fatal flaw. It had no control group and, therefore, cannot say conclusively that Compound Q's effects were any better than those of a placebo.
Still, the apparent magnitude of the results, and the fact they were delivered at one of the meeting's major scientific forums, drew new attention to a drug that has been the subject of intense interest.
"At this stage," said study director Martin Delaney, "we have seen a major and dramatic change in what happens to the patient. "We do not see anything like this with AZT." Delaney is executive director of Project Inform, the activist group that conducted the trial.
Delaney's talk drew immediate reaction.
"There is no way on earth to determine from the data whether this is a flash in the pan or something great," said Arnold Relman, editor of the New England Journal of Medicine.
Relman called Delaney's decision to release the results of the study "irresponsible" and likened the study's design, without a control group, and its limited review by other scientists, to "black magic."
"Neither physicians nor patients should make any decisions based on this information until it's been thoroughly reviewed," said Daniel Hoth, director of AIDS clinical trials at the National Institutes of Health.
The controversy is the latest in a series of disputes over the drug, an extract from the roots of a Chinese cucumber plant that some researchers and many AIDS patients are convinced shows potential in slowing the progression of HIV infection.
Last year, one patient being treated in an underground clinical trial of Compound Q died, causing widespread criticism of the unauthorized experiments and triggering a Food and Drug Administration investigation. The debate over the drug also demonstrates the continuing tension between the desire of the scientific community to conduct formal, controlled trials of promising drugs before approving them for widespread use and the desire of HIV-infected people to gain access to potential remedies as quickly as possible.
Compound Q, which has been used as an abortifacient in China for centuries, first came to the attention of the AIDS community when laboratory experiments showed it to be highly effective in killing cells infected with HIV.
Other data presented here today, for example, shows that Compound Q in the laboratory kills the immune system cells known as macrophages that are infected with HIV, while sparing normal cells.
Some researchers suspect that killing macrophages is critical to stopping AIDS because the cells, unlike other infected immune system cells, do not die soon after infection, but linger to serve as a reservoir for the virus.
These results, however, are also controversial because a number of other laboratories have been unable to duplicate them.
The Project Inform study, which began last November with 46 patients, focused on the effect Compound Q had on the immune system's T cells. The defining feature of HIV infection is a steadily declining number of T cells.
The patients in the Project Inform study had been slowly losing T cells, but after four monthly injections of Compound Q, the average number of T cells in the group rose significantly.
It is known that psychological factors can alter the immune system's effectiveness and, critics of the study note, the patients knew they were receiving an experimental drug.