Publication Type Review
Authors Arkin L, Sondhi D, Worgall S, Suh L, Hackett N, Kaminsky S, Hosain S, Souweidane M, Kaplitt M, Dyke J, Heier L, Ballon D, Shungu D, Wisniewski K, Greenwald B, Hollmann C, Crystal R
Journal Hum Gene Ther
Volume 16
Issue 9
Pagination 1028-36
Date Published 09/01/2005
ISSN 1043-0342
Keywords Genetic Therapy, Motivation, Neuronal Ceroid-Lipofuscinoses, Patient Acceptance of Health Care, Patient Selection
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.
DOI 10.1089/hum.2005.16.1028
PubMed ID 16149901
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