Study Rationale: New therapies for Parkinson’s disease (PD) that involve the use of cells or genes — sometimes called advanced therapy medicinal products (ATMP) — are being developed and tested in clinical trials. These studies, which involve large numbers of volunteers and last years, can be very challenging for participants. We have interviewed individuals several years after they’ve participated in such studies as part of a project called LEARN (Listening to the Experience of pARticipants on Neurosurgical trials). Now, we will extend our approach to people actively participating in an ATMP trial for PD to better understand their experience.
Hypothesis: We believe that by learning more about what participants experience during trials of cell therapy, and talking more widely to the PD community, we can improve ATMP trials by providing guidance to the investigators designing the studies and to the individuals taking part.
Study Design: We will interview the participants and care partners of the STEM-PD stem cell trial which will soon begin in the UK and Sweden. Participants will be interviewed before and during the trial. Additionally, to understand what people think about cell and gene therapies in general and evaluate why there is limited diversity across trial participants, we will also survey the wider PD community. Some survey respondents will be followed up with more in-depth interviews.
Impact on Diagnosis/Treatment of Parkinson’s disease: Our results will be used to develop a framework for improving the design and conduct of ATMP trials. We will identify ways to keep participants engaged in these lengthy studies and to make the trials more widely accessible to the PD community, thereby facilitating the availability of effective treatments.
Next Steps for Development: We will use data from this study to generate strategies to encourage more diverse and inclusive participation in cell and gene therapy trials.