When a scientist petitions for the right to use gene therapy in a human being, theoretically every American will have a chance to comment on the proposal before the National Institutes of Health's Recombinant DNA Advisory Committee.
Known as RAC, the committee is part of a multi-tiered review process established to help ensure the safety of the first human gene therapy experiments.
"Just as we have learned not to leave war solely to the generals, scientists alone should not control human gene therapy," writes Judith Areen, associate dean of Georgetown University Law School, in an upcoming West Virginia Law Review article.
"New areas of work require new review processes," adds LeRoy Walters, chairman of the Working Group on Human Gene Therapy, an advisory committee to RAC, and head of the Center for Bioethics at Georgetown University.
The review process for a human gene therapy experiment begins at the local level -- at the hospital or medical center where the treatment will be performed. There, groups known as institutional review boards and institutional biosafety committees must approve the proposal, or "protocol," based on federal guidelines established by the Department of Health and Human Services.
The regulations require that there be one lawyer, one ethicist or member of the clergy, and at least one person from outside the institution sponsoring the research. These panels also must have both men and women among their five members.
The gene therapy proposal then moves on to the national level -- to the Working Group on Human Gene Therapy, a 14-member NIH advisory committee whose membership must include four physicians, two microbiologists, three lawyers, three ethicists, two public policy experts and one average citizen.
The Working Group wrote, and RAC recently approved, "Points to Consider" -- a set of guidelines for scientists interested in doing gene therapy.
Next the proposal for gene therapy goes to RAC and finally to the NIH director, Dr. James Wyngaarden. The proposal also will be simultaneously submitted to the Food and Drug Administration for approval.
Especially during the early years of gene therapy experiments, says Dr. Walters, "it is important to have a public national review process. We hope to set up regular schedule of three annual meetings of RAC and the working group, at four month intervals."
The hope, he says, is to avoid national controversy over new medical technology such as the uproar created during the Baby Fae case, in which a baboon's heart was implanted in an infant. That incident, Walters says, "illustrates the importance of having someone from outside the institution doing the review work. The hospital ethics committee, the institute's review board and the whole institution were caught up in the enthusiasm."
"It will not be easy to decide what limits, if any, to place in the path of human gene therapy," says Georgetown's Areen, who is also a Working Group member. "At a minimum, we must remain vigilant about the dangers of false cures."
A bill before Congress, introduced by Sen. Albert Gore (D -- Tenn.), would establish a 12-member congressional Bioethics Board modeled after the Office of Technology Assessment. The board would be bipartisan and would appoint yet another advisory board of scientists, physicians, medical ethicists, lawyers, clergy and lay people.
"On some occasions in the past, we have seen revolutionary developments in science overwhelm the analytical capacity of free society to make the choices inherent in scientific developments," Gore says. "If we had taken more time to comprehend the implications of nuclear technology when it was created, perhaps we would have developed a keener appreciation for the choices we had to make. And perhaps we might have found the way to avoid the situation we've created."
Genetic engineering, including gene therapy, "will almost certainly have an enormous impact on human society," he says. "We can see that impact, we can see it coming. We should accelerate our efforts to define the choices that will soon confront us, and the sooner that begins the better, because technology can develop a momentum of its own . . .
"We make the assumption that somehow science will work everything out for us and everything will come out all right in the end. But it won't necessarily. And we have the ability to choose good effects and avoid bad effects and not surrender to the technological imperative that's inherent."