Examining the viewpoints and decision-making process of Sickle Cell Disease patients, parents, and physicians towards prospective participation in CRISPR-Cas9 clinical trials

Authors

• A Persaud
• BA
• VL Bonham
• JD

Abstract

The advent of genome-editing technologies, specifically CRISPR/Cas9, implicates both tremendous therapeutic promise and significant potential risk. Because sickle cell disease (SCD) is a well-studied molecular disorder impacting the blood system, it is thought to be an ideal candidate for gene-editing therapies. However, there is limited research on patient, parent, and physician perspectives, within this community, towards clinical research related to the use of gene-editing technologies. Here we explore the knowledge, values, belief systems, and decision-making process of these three populations within the context of prospective clinical trial enrollment. This is a qualitative mixed-methods study of individuals from the Southern and mid-Atlantic regions. It involves survey data and moderated, audio-recorded focus groups. In the preliminary analysis, we identified emerging themes around hypothetical CRISPR-based trial participation from 9 focus groups. Clinical trial-exogenous stressors and involvement burden, stage of life (age) of prospective participant, the capacity of gene-editing to improve quality of life, an assessment of risk and benefit, especially related to off-target events, long-term consequences, and the sustainability of the therapy, were dominant factors in the decision-making process to participate/recommend participation for all three groups. Two other major themes, found across the patient/parent focus groups, but absent from the physician groups, include the potential to help others with SCD, as well as one’s spiritual beliefs and relationship with God. The focus group participants also explored the types of information desired before making a decision to participate/recommend participation in a gene-editing trial, as well as credible sources of this information.

Scientific Focus Area: Social and Behavioral Sciences

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