Community Research Seeks to Speed Work on AIDS Treatments
The head table at a banquet here Friday evening featured some of the federal government’s top health officials: Dr. Anthony Fauci, AIDS research chief of the National Institutes of Health; Dr. Samuel Broder, director of the National Cancer Institute, and White House physician Dr. Burton Lee.
But even more remarkable was the identity of their host: Michael Callen, who has AIDS himself and has been a frequent critic of the pace and priorities of the federal government’s AIDS research program.
“After a lot of shouting, we have all decided we have more to gain from listening to one another,” said Callen, who is president of the board of governors of New York’s Community Research Initiative. Added Lee, who served on President Reagan’s AIDS Commission: “It is a marriage between the brain trust and the people in the trenches.”
The dinner was part of a three-day conference on community-based AIDS research, an emerging movement that is harnessing the energy and expertise of local physicians throughout the country in the search for effective AIDS treatments. Once viewed skeptically by researchers in academic medical centers, community-based research proved its value earlier this year when the U.S. Food and Drug Administration approved the use of aerosolized pentamidine to prevent AIDS-related pneumonia based on data gathered by community groups in San Francisco and New York.
A year ago, those were the only two cities with operating community-based research organizations. This weekend’s conference included representatives of 16 such new research groups from throughout the country who came to learn the nuts and bolts of performing clinical drug trials.
Dr. Mathilde Krim, founding chair of the American Foundation for AIDS Research, said a functioning network of community-based research groups is “absolutely essential” to help test “the new crop” of drugs now emerging from laboratories.
Without such a network, she warned, “a rich harvest of research advances will remain unexplored, inapplicable and unused while the toll of death and despair will continue to rise, horribly, all around us, month after month, year after year.”
The nation’s total number of reported AIDS cases is approaching 100,000 and will reach 285,000 by the end of 1991, according to the U.S. Centers for Disease Control’s “best estimate.” The General Accounting Office predicts that up to 485,000 Americans will be diagnosed by that time.
The new network should greatly expand access to experimental medications, especially in regions that lack academic AIDS treatment research centers. “This could give our community a shot at DDI or DDC,” two promising experimental antiviral drugs, said Dr. Melanie Thompson of the Atlanta AIDS Research Consortium. Atlanta AIDS patients previously had to go to Miami or Durham, N.C. to participate in clinical trials.
The weekend gathering indicated that, while key questions remain unsettled, a new consensus on how to speed access to experimental drugs for AIDS and the underlying human immunodeficiency virus is emerging. The key elements of the new system include community-based trials as an adjunct to university-based studies, as well as expanded access to experimental drugs under Fauci’s just-unveiled “parallel track” initiative. The initiative would permit patients who are unable to enroll in efficacy trials to obtain the drug under certain circumstances.
Despite the fears of some advocates of regulatory reform, last month’s disclosure that a patient had died during the underground trials of a touted Chinese drug known as Compound Q does not appear to have resulted in any backlash against the current movement toward liberalization.
Indeed, advocates of swifter patient access to promising drugs were as insistent as ever. “Our job is not to study new therapies,” said Broder, a developer of AZT, the only approved antiviral drug for AIDS. “It is to bring new therapies into medical reality. We must make sure that our governmental apparatus can develop drugs, and then get out of the way.”
Lee, who was a cancer researcher at Memorial Sloan Kettering Hospital here before he became President Bush’s personal physician, was even more outspoken. “I hope you bring down a large part of this drug regulatory system that has been built up over the last 30 years,” he said in his remarks to the community researchers. “I love to see power going back to the people.” Added Fauci: “What I see in community-based research is totally compatible with the mission of the National Institutes of Health.” The NIH is providing $6 million for community-based trials this year and has budgeted a similar sum for next year.
Mark Harrington, a member of the advocacy group ACT UP, said community trials will be particularly useful in testing the efficacy of drugs to control the opportunistic infections that characterize AIDS. Most academic research focuses on finding treatments to stop the underlying virus, HIV. Community trials, he said, “will be geared not toward finding some magic bullet against HIV five years from now, but toward keeping patients alive today.”
Fauci said his proposed “parallel track” program would “allow individuals who were otherwise disenfranchised to get some form of therapy.” These would be individuals disqualified from participating in more rigid clinical trials which, he noted, are exclusionary by their very nature.
Drug Makers’ Concerns
Krim, however, warned that pharmaceutical companies might be reluctant to supply drugs for the parallel track because of the costs involved.
And Dr. Ellen Cooper, director of the FDA’s division of antiviral drugs, said she is “concerned” that Fauci’s pronouncements had raised patient’s expectations about rapid drug release “unrealistically.”
Still, in a an indication that the agency is liberalizing its efficacy standards for AIDS drugs, she said the FDA will consider a wide range of laboratory and clinical parameters in deciding whether to license a drug against AIDS. In the past, the agency has evaluated a drug’s efficacy by comparing the number of deaths of patients taking the drug in question with the number of deaths in a control group.