Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders

Lisa M. Arkin, Dolan Sondhi, Stefan Worgall, Lily Hyon K. Suh, Neil R. Hackett, Stephen M. Kaminsky, Syed A. Hosain, Mark M. Souweidane, Michael G. Kaplitt, Jonathan P. Dyke, Linda A. Heier, Douglas J. Ballon, Dikoma C. Shungu, Krystyna E. Wisniewski, Bruce M. Greenwald, Charleen Hollmann, Ronald Crystal

Research output: Contribution to journalReview article

27 Citations (Scopus)

Abstract

Genetic medicine-based therapies have unlocked the potential for ameliorating diseases previously considered inevitably fatal. Inherent in the clinical trials of genetic medicines are ethical issues of therapeutic misconception, enrollment decisions as they relate to the risks and benefits of research, and the complex relationships among funding sources, investigators, and the families of affected individuals. The purpose of this paper is to help define these complex issues relevant to the use of genetic medicines and to describe the strategy we have used to confront these issues in a phase I trial of adeno-associated virus-mediated gene transfer to the central nervous system of children with late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal lysosomal storage disease associated with progressive neurodegeneration and death by mid-childhood. Our approach to these challenges should provide a useful paradigm for investigators initiating other genetic medicine-based studies to treat inevitably fatal diseases.

Original languageEnglish
Pages (from-to)1028-1036
Number of pages9
JournalHuman Gene Therapy
Volume16
Issue number9
DOIs
Publication statusPublished - 1 Sep 2005
Externally publishedYes

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ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

Cite this

Arkin, L. M., Sondhi, D., Worgall, S., Suh, L. H. K., Hackett, N. R., Kaminsky, S. M., Hosain, S. A., Souweidane, M. M., Kaplitt, M. G., Dyke, J. P., Heier, L. A., Ballon, D. J., Shungu, D. C., Wisniewski, K. E., Greenwald, B. M., Hollmann, C., & Crystal, R. (2005). Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders. Human Gene Therapy, 16(9), 1028-1036. https://doi.org/10.1089/hum.2005.16.1028