One would think that the fallout from the public relations disasters of the past year in agbiotechnology would have alerted gene therapists to the importance of allaying public anxieties concerning the risks (whether perceived or real) of their technology. Instead, judging by comments in some quarters, there appears to have been complacency and even irritation at the intense publicity surrounding the death of an 18-year-old patient in trials last year.

Some of this appears to stem from a (somewhat justified) feeling that the two overseeing regulatory agencies—the US National Institutes of Health's Recombinant DNA Advisory Committee (RAC) and the Food and Drug Administration (FDA)—should set their houses in order and provide clear guidelines for the disclosure of adverse events in trials. But it also appears to originate from a perception that the increased scrutiny of regulatory agencies and the public at large is a nettling development that will stifle progress of research and commercial development of the technology.

Some positive things have come from this unfortunate affair. For one thing, James Wilson, who heads the Institute for Gene Therapy, where the trial was carried out, has emerged with considerable dignity. In addition, the RAC (as well as the FDA) has now clarified its requirement for notification of all serious adverse events.

On the other hand, several notable researchers have revealed a disconcerting lack of awareness of the importance of public confidence for ensuring the future success and acceptance of gene therapy. For example, at a press briefing during the recent “Biology of Drug Discovery” symposium at the Whitehead Research Institute, a high-profile researcher denied that the gene therapy deaths were a cause for major concern, asserting that research should continue as normal. Apparently, the only message to be taken from the whole ruckus was that there is still a lot of basic biology to learn when it comes to how the body responds to foreign genetic material.

In November, at the Nature Biotechnology Gene Therapy symposium in Washington, a representative from a large multinational pharmaceutical company was overheard railing about the inexperience of the RAC in overseeing clinical trials. The implication clearly was that the blame for the recent bad press lay with RAC, not with the reluctance of corporate researchers to disclose adverse events.

With the prospect of new technologies and vectors coming to the clinic in the near future, gene therapists must accept the interest of the public in their technology. These are experimental technologies and knowledge of the basic biology is still evolving; little wonder, then, that the regulatory framework is less than perfect and also evolving.

Researchers must not only accept the right of regulators to scrutinize their protocols, but also acknowledge the importance of open public debate on the benefits and risks of gene therapy. To do anything less risks a loss of public confidence. And once public confidence has been lost, it cannot easily be recaptured.