Volunteering for early phase gene transfer research in Parkinson disease.
BACKGROUND
For early phase trials of novel interventions-such as gene transfer for Parkinson disease (PD)--whose focus is primarily on safety and tolerability, it is important that participants have a realistic understanding of the goals of such research. Recently, some have expressed concern that patients with PD may have unrealistic expectations.


METHODS
The authors examined why patients with PD might volunteer for invasive early phase research by interviewing 92 patients with PD and comparing those who would (n = 46) and those who would not (n = 46) participate in a hypothetical phase I gene-transfer study.


RESULTS
The two groups' demographic, clinical, functional, and quality of life measures, as well as their understanding of the research protocol, were similar. The groups did not differ on their perception of potential for personal benefit nor on the level of likelihood of benefit they saw as a precondition for volunteering. However, those willing to participate tended to perceive lower probability of risk, were tolerant of greater probability of risk, and were more optimistic about the phase I study's potential benefits to society. They also appeared more decisive and action-oriented than the unwilling group.


CONCLUSIONS
It is likely that the decision whether to participate in early phase PD gene transfer studies will depend mostly on patients' attitudes regarding risk, optimism about science, and an action orientation, rather than on their clinical, functional, or demographic characteristics.
Kim
S Y H
SY
Department of Psychiatry, Bioethics Program, Center for Behavioral and Decision Sciences in Medicine, University of Michigan, Ann Arbor, USA. scottkim@umich.edu
Holloway
R G
RG
Frank
S
S
Beck
C A
CA
Zimmerman
C
C
Wilson
R
R
Kieburtz
K
K
D001294:Attitude to Health; D018014:Gene Transfer Techniques / Q000523:psychology*; D015316:Genetic Therapy / Q000523:psychology*; D006805:Human Experimentation; D006801:Humans; D007258:Informed Consent; D010300:Parkinson Disease / Q000235:genetics* / Q000628:therapy*; D011788:Quality of Life; D018570:Risk Assessment