Volunteering for early phase gene transfer research in Parkinson disease
S Y H Kim1, R G Holloway, S Frank
1Department of Psychiatry, Bioethics Program, Center for Behavioral and Decision Sciences in Medicine, University of Michigan, Ann Arbor, USA. scottkim@umich.edu
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.
Insights
Patient decisions for early phase Parkinson disease (PD) gene transfer trials hinge on risk perception and optimism, not clinical factors. Understanding these attitudes is key for realistic participant expectations in novel PD research.
Area of Science:
- Neuroscience
- Clinical Trials
- Genetics
Background:
- Early phase trials for novel Parkinson disease (PD) interventions, like gene transfer, prioritize safety and tolerability.
- Ensuring participants have realistic expectations is crucial for ethical research conduct.
- Concerns exist regarding potentially unrealistic patient expectations in PD research.
Purpose of the Study:
- To investigate factors influencing patient decisions to volunteer for invasive, early-phase gene transfer research in Parkinson disease.
- To compare the characteristics of PD patients willing to participate versus those unwilling in a hypothetical Phase I gene transfer study.
Main Methods:
- Interviewed 92 patients with Parkinson disease (PD).
- Compared 46 participants willing to join a hypothetical Phase I gene transfer study with 46 unwilling participants.
- Assessed demographic, clinical, functional, quality of life, and understanding of research protocol.
Main Results:
- No significant differences were found in demographic, clinical, functional, or quality of life measures between groups.
- Both groups had similar perceptions of potential personal benefit and its precondition for volunteering.
- Willing participants perceived lower risk, tolerated higher risk probability, showed more optimism about societal benefits, and were more action-oriented.
Conclusions:
- Participation in early-phase PD gene transfer studies is primarily influenced by attitudes towards risk, scientific optimism, and action orientation.
- Clinical, functional, or demographic characteristics are less likely to determine participation decisions.
- Understanding patient attitudes is vital for managing expectations in PD gene therapy trials.
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