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

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