First-In-Human Stem Cell Trials in Huntington’s Disease: A Bioethics Survey (S25.008)

Objective: Explore attitudes and concerns of Huntington’s disease community about participation in a first-in-human experimental approach to treatment of HD. Background:Experimental treatment approaches and first-in-human Phase 1 trials are ethically complex. These studies create challenges for informed consent, impose burdens and risks to subjects and may offer little or no prospect of clinical benefit. Similarly, these studies create bioethical concerns for investigators, coordinators and study staff. These issues are understudied in the HD population. Methods: An anonymous survey approved by the Institutional Review Board at UC Davis. Results: Respondents (209/268) were separated into either Group 1: Individuals at risk or genetically tested for HD (n = 116) or Group 2: Family members or care providers (n = 93). Respondents were largely Caucasian (96[percnt]), and more likely female (69[percnt]), with mean age of 52 years (range 18 - 79) and drawn from throughout the US. Regarding participation in a first-in-human trial for HD, 67[percnt] of individuals from Group 1 responded positively compared to 92[percnt] from Group 2. For Group 1, most respondents reported no ethical concerns regarding experimental approaches to the treatment of HD. The most important reason cited for considering participation was to find a cure for HD. The perceived burdens of participation in a first-in-human trial included concerns about access and fear of adverse effects. For Group 2, sixty-five percent of respondents had no ethical concern regarding experimental approaches, with 57[percnt] supporting participation in such a study. Group 2 respondents were more likely to express safety concerns and adverse events than were those from Group 1. Perceived burdens related to participation were logistical, psychological and physical risk. Further statistical analyses will be presented. Conclusions: An appreciation for ethical challenges may impact study design and enable investigators and participants to bridge communication gaps to enhance the process of informed consent. Disclosure: Dr. Duffy has nothing to disclose. Dr. Tempkin has received personal compensation for activities with Lundbeck Pharmaceuticals. Dr. Martin has nothing to disclose. Dr. O9Keefe has nothing to disclose. Dr. Michie has nothing to disclose. Dr. Yarborough has nothing to disclose. Dr. Wheelock has nothing to disclose.