A Matter of Trust
Authors: Fintan R. Steele
Journal: Molecular Therapy (2000)

Public trust in gene therapy and therapists, if not shattered, is seriously fractured. The hard truth that there are still no genetic magic bullets has dawned on almost everyone, save a few proponents who continue to test the shaken public trust by their ill-advised and even specious pronouncements of imminent triumph. Can the fracture be mended? Yes, but it will require difficult and vigilant effort. The fracture lines follow two directions. The first is the increased difficulty in fully enrolling current clinical trials. Although no hard data are available, almost everyone now enrolling trials offers anecdotal evidence that potential volunteers are fewer, and those who do express an interest are more hesitant to commit for fear of personal tragedy. If this trend continues, it could help push eventual success even further into the future. The second fracture line is, perhaps, far more serious. Recent adverse events have been interpreted to an already skeptical public through mass media stories that inaccurately focus on unrevealed conflict of interest, lack of adherence to safety regulations, and the egotistical drive for personal fame and fortune at the expense of patients. This media-painted patina of scarlet colors the public perception of all gene therapies and therapists and is pushing public skepticism into public cynicism. What can we do? Frankly, much of the answer to that rests on the personal integrity of the scientists running trials. However, there are specific repairs we can agree upon as a group to undertake now. In regard to enrollment problems, it is clear that potential trial volunteers need more information, not less. The idea of a “patient advocate,” raised by Jesse Gelsinger's father, is worth serious consideration, although the title is probably not the best. All relevant information, including that known and unknown, must be shared with the patient and his/her supporters. The scientific context of any given trial must also be provided. Granted, this raises almost as many problems as it solves, but they are far less serious problems than those dealing with the chaos that follows a serious adverse event that might have been foreseen but which was undisclosed. We must go farther and ask ourselves if all the current trials that are having trouble enrolling should even be performed. Many will say off the record that there are trials planned or underway for which the scientific basis is dicey at best and may even support halting the trial. Ideally this decision should come from the sober judgment of the investigator leading the trial. Nevertheless, it may be time to reconsider the mechanism for passing scientific judgment (e.g., a scientific peerreview nongovernmental group, a role that should be played by the local IRB but is sadly not always fulfilled). What can we do about the more difficult issue of the alarming public perception that gene therapy is awash in either inept researchers or those out only for personal gain? Granted, we don't control the press, but neither do we all spend the time we need to in ensuring that they get the whole story, negative and positive. Ways to address this are already being discussed by the ASGT and elsewhere, and it is hoped this will lead to some remedies. Indeed, a meeting of several academic and industrial gene therapists is underway as this issue goes to press. The results of their grappling with these problems will appear in the May issue. For our part, Molecular Therapy is now requiring that every author reveal any potential or real conflict of interest or conflict of commitment before a paper will be considered for publication. These conflicts will not always be published, but if the editors believe them relevant, they will appear in the “acknowledgements” of a published manuscript. We strongly believe that restoring at least part the public trust relies on reestablishing the openness that characterizes the best of scientific and medical advances.