Gene Therapy and Jurisprudence
Authors: Inder M. Verma
Journal: Molecular Therapy (2001)

Imagine parents sitting in a physician's office learning about a new treatment for their child's rare disease. The treatment is effective but costly, and is not covered by any healthcare provider. The treatment is gene therapy and as there are no previous medical options, no treatment costs were set. The third-party provider will undoubtedly resist payment, leading to disputes and, eventually, the courts. Early this past July, marooned on the tiny volcanic island of Hawaii, I attended “The Courts’ First International Working Conversation on Enviro/Genetics: Disputes and Issues.” The attendees were not my usual colleagues— molecular biologists, virologists, vectorologists, or clinicians—but a group of international jurists with awe-inspiring titles like “Chief Justice,” “Justice,” “Session Judge,” and “Federal or Circuit Court Judge,” all with the formidable prefix or suffix of “Honorable,” “Esquire,” and so on. The jurists were eager to learn about the new technologies invading their courts in increasing numbers, ranging from forensic science, patents, genetically modified organisms (GMO) and food, transgenic animals, cloning and stem cells to, above all, gene therapy. They were curious about gene delivery systems, choice of disease, patient selection and care, costs, conflicts of interest, consent forms, and a host of related issues. Most of all, they wanted to be educated about the science behind these new modalities. Gene therapy fascinated them because of its potential for treating previously incurable diseases and for slowing down the progression of deadly or debilitating diseases. They had five main concerns. Consent forms. Are patients sufficiently informed to enroll in gene therapy trials? Who protects their interests? To what extent do the parents have authority to enroll a child in a gene therapy trial? Cost and Patient Care. The courts are concerned that this area is rife with conflict because such novel therapies may not be mentioned in health policies (Providers function on the doctrine of “expressio unius est exclusio alterius,” which holds that when a list includes specific items, items not included are presumed to be excluded.) Who will provide for the patient's long-term care or follow-up visits? What if a superior treatment comes along—will providers renege? Conflict of Interest. Does every institutional or commercial concern have a generally acceptable conflict-of-interest policy that is available to the patient and the public? Jurists want clear guidelines on the issue of enrollment of patients by individuals or outfits that may commercially benefit from the outcome of the therapy. Genetic Enhancement. Every parent wants to give their offspring the best chance in life, but will they resort to genetic enhancement to gain significant physical or social advantage? The courts are worried that they will have to struggle with the issue of legal limitations to parents’ authority to manipulate genetic characterizations of their children. Malpractice. The circumstances of Jesse Gelsinger's death following gene therapy vector administration fascinated them. I was peppered with questions from “Was it the right disease?” to “Was there enough supervision by the appropriate regulatory agencies?” I got the impression that most of the jurists would like to have seen a fullblown trial rather than the University of Pennsylvania settling out of court. They were hungry for details and precedent, which I could not supply. We are entering uncharted territory, where science and law will clash and cooperate to set the standards of the society in which we live. The law is no stranger to controversy and imbalances between interacting parties. A meeting between scientists and jurists is a step in the right direction: A well-informed jurist can make a more considered decision.