H U M A N G E N E T H E R A P Y 2:235-242 (1991) Mary Ann Liebert, Inc., Publishers

Liability C o n s i d e r a t i o n s P r e s e n t e d b y H u m a n





Through the use of a hypothetical scenario, this article examines the legal liability associated with gene therapy. Basic negligence principles are applied to the factual context of a h u m a n gene therapy experiment gone awry, including its prior governmental review and its potential effect on future generations. T h e federal requirements, while not preempting state law damages claims, do provide a mechanism for achieving some protection from liability. T h e effect on future generations raises questions about the limits of liability.


us uncomfortable, it is the best backdrop for the discussion that follows. Now that human gene therapy is into clinical trials, theSuppose that, while gene therapy technology is still in its question of liability is being raised. W h o can be held liable if infancy, Researcher "Ron" develops a potential somatic gene something goes wrong? H o w vulnerable are investigators, therapy for a rare genetic disease called "Empo," which is institutions, and the companies that provide material? caused by a deletion in the "Emp" gene. The therapy involves Palmer, a practicing attorney with considerable knowledge autologous bone marrow transplant using retroviral vectors of the science and practice of gene therapy, examines these containing the wild type E m p gene. The proposed procedures have proven successful in animal experiments. Ron works at questions. "Dodge," a large research institution that receives federal funds, along with a physician named "Paul," who treats a small population of patients suffering from Empo. INTRODUCTION Ron and Paul write a protocol for a human gene therapy experiment, carefully following the National Institutes of Health's (NIH's) "Points to Consider in the Design and SubmisWhen human gene therapy was merely a "fantastic" theory, the legal liability associated with it did not sion of Protocols for the Transfer of Recombinant D N A into the generate much concern. As gene therapy emerges from theory Genome of Human Subjects" (Points to Consider). Ron and Paul into reality, physicians, biotechnology companies, and health plan to perform the human experiment at Dodge, using vectors care institutions should begin to consider their exposure to provided by Company "Coe,' the research sponsor. They submit their protocol to Dodge's institutional review board and liability for potential unplanned consequences. This article will explore some of the legal issues facing institutional biosafety committee, both of which approve it after providers of human gene therapy, using the following hypothet- thorough examination. Ron and Paul also submit their protocol ical situation. The hypothetical presents a "worst-case" sce- to the NIH Recombinant D N A Advisory Committee's (RAC's) nario, involving a highly unlikely combination of several im- Subcommittee on Human Gene Therapy (Subcommittee) and to probable events. Although one could imagine a more realistic the Food and Drug Administration (FDA). A summary of the scenario (e.g., one in which gene therapy causes cancer in a protocol is published in the Federal Register and subjected to patient), such a scenario would not present as many significant public comment. After many months, many intensive reviews legal issues. Indeed, the most unlikely events and the most and several revisions, the protocol is approved. It has been unforeseen circumstances have sparked some of the greatest scrutinized and approved by seven committees and discussed in legal debates. While this "worst-case" hypothetical might make several public forums.

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236 Ron and Paul treat six human subjects with their experimental gene therapy. All of the human subjects are unmarried, young adults and all have received extensive information about the risks and benefits of the experiment as well as the alternative treatments, in accordance with federal regulations. All have signed consent forms. The results of the experiment are overwhelmingly positive, at first. All of the patients recover from E m p o and m o v e on to lead normal lives. Five years later, one of the former research subjects, Sally, has a child named Ben w h o suffers from severe limb deformities. A s a result, Sally suffers terrible emotional distress. Upon investigation, it is discovered that an unintended complication of the gene therapy experiment caused Ben's deformities. The retroviral vector containing E m p recombined with an endogenous retrovirus in Sally and infected cells throughout her body, including some of her ova. Ben's limb deformities were caused by an abnormality in a protein which is involved in fetal limb development. Laboratory tests identify a chimeric protein that is comprised of this developmental protein fused with the E m p gene product. A prenatal examination of another former patient from the same protocol reveals the same problem in her fetus/[) Sally and Ben (the plaintiffs) sue Ron, Paul, Dodge, and Coe (the defendants). Sally and Ben claim that (i) the defendants were negligent in not informing Sally of the specific risk that she might have a child with limb deformities; (ii) the defendants negligently employed the vector in humans before it had been tested in animals for a sufficient period of time; and (iii) the defendants were negligent in using a nonstandard medical procedure to treat Sally. Sally and Ben seek damages for the cost of extraordinary medical, educational, and other care that Ben will need. Sally also seeks damages for her mental anguish and emotional distress. Ben seeks general damages for his impairments.

2. The patient must prove that the health care provider "breached" that duty, i.e., failed to satisfy the "standard of care." 3. The patient must prove that the health care provider's breach of duty caused the patient's injuries.(2) 4. The patient must prove the existence and amount of legally recognized damages.(3) Each of these four requirements apply to Ben and Sally.

APPLICATION OF T H E LEGAL PRINCIPLES T O G E N E THERAPY Federal preemption of state law damages claims

An initial legal question is whether the extensive federal involvement with gene therapy precludes, or "preempts," Sally's and Ben's state law-based claims. Preemption is based on the United States Constitution's supremacy clause, which makes Federal law the "supreme law of the land."(4) Where the United f States Congress has declared that it is expressly preempting state law, where preemption can be implied from a statute, or where state law actually conflicts with federal law, state regulation of a given area is precluded. (Tribe, 1988, § 6-26, n. 14).(5) Ron, Paul, Dodge, and C o e might argue that Sally's and Ben's claims should be dismissed because they are impliedly preempted by the extensive Federal requirements the defendants had to meet before their gene therapy protocol was approved.

Liability considerations presented by human gene therapy.

Through the use of a hypothetical scenario, this article examines the legal liability associated with gene therapy. Basic negligence principles are ap...
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