Patients’ Willingness and Perspectives Toward Chimeric Antigen Receptor T-Regulatory Cell Therapy for Inflammatory Bowel Diseases

• Published in: Crohn's & colitis 360 2020-10, Vol.2 (4), p.otaa085-otaa085
• Main Authors: Vent-Schmidt, Jens, Goldsmith, Laurie J, Steiner, Theodore S
• Format: Article
• Language: English
• ISSN: 2631-827X; 2631-827X
• DOI: 10.1093/crocol/otaa085

Abstract Background Inflammatory bowel disease is a life-changing disease resulting from recurrent intestinal inflammation. Current therapies (eg, steroids and biologics) are associated with mild to severe side effects, and none provide a cure. Recent research has focused on genetically engineering gut-specific anti-inflammatory T-regulatory cells (CAR-Tregs) to control intestinal inflammation, a logistically and conceptually complex approach. The purpose of our study was to understand patients’ willingness to try CAR-Treg given 2 hypothetical scenarios—in a clinical trial or as a new treatment. Methods We surveyed people living with inflammatory bowel disease about their willingness to try CAR-Treg. The online survey was developed using patient focus groups and associated literature. We recruited participants through email and social media. We used descriptive and inferential statistics to analyze closed-ended questions and inductive thematic analysis to analyze open-ended follow-up questions. Results Survey participants indicated high willingness to try CAR-Treg therapy in both a clinical trial and as a new treatment. Willingness to try was not correlated with disease state or medication history. Women were less likely than men to indicate willingness to participate in a clinical trial. Participants’ reasons for being willing to try CAR-Treg therapy included the wish to change their current treatment and the calling to participate in research. Participants that were not willing to try CAR-Treg mentioned the lack of long-term data and the success of their current therapy. Conclusions This is the first study to our knowledge to investigate patient willingness to try CAR-Treg therapy. Our results demonstrate the promise of moving this therapy into clinical practice as most patients indicated willingness to try. Lay Summary We surveyed people living with inflammatory bowel disease about their willingness to try a new therapeutic approach currently developed in several research laboratories—chimeric antigen receptor-expressing T-regulatory cells—and found high willingness to try, independent of disease state or medication history.