VOLUME 28, ISSUE 2 • JUNE 2024. Full issue »
Conference focuses on trial design for PD prevention
The ‘2024 Planning for Prevention of Parkinson's: A Trial Design Forum’ was in Boston from May 5-7. The conference, organized by Massachusetts General Hospital in collaboration with the Parkinson Study Group and the International Parkinson and Movement Disorder Society, brought together key stakeholders in Movement Disorders from advocacy, academia, foundation, industry, government, and regulatory organizations to discuss the developments of the first Parkinson’s disease prevention trial.
The initial agenda included an interactive walk discussing the role of exercise led by Dr. Bas Bloem, and a Boston Harbor boat tour and talk discussing direct-to-consumer testing for PD moderated by Dr. Poston. Subsequent sessions highlighted the challenges of prevention trials in Parkinson’s disease and specific pragmatic strategies to address them. The breakdown of sessions included who to test, what to test, and how to measure specific outcomes. Key discussions suggested that individuals at risk for PD are more likely to be included, including those with genetic forms of PD, as well as positive biomarkers, including DatScan and alpha-synuclein seeding assays.
The conference organizers, Dr. Grace Crotty from Cork University of Hospital and Dr. Michael Schwarzschild from Massachusetts General Hospital, highlighted the importance of the conference: “This conference provides a pre-competitive space for the free exchange of early PD prevention trial protocols. And as concepts of biological definition are evolving to encompass a broader set of synucleinopathy-linked neurodegenerative diseases, our conference series is bringing together the dementia with Lewy bodies and PD research communities, given the parallel prospects for prevention of these diseases when targeting key at-risk populations. In our conference, we focused on secondary prevention trials where the goal is to stop or delay the development of traditionally diagnosed PD (e.g., as defined by current clinical diagnostic criteria centered on motor deficits) during the prodromal period of the disease.”
Among the many highlighted sessions, an engaging discussion compared the two new proposed classification systems for Parkinson disease, with the participation of Dr. Tony Lang and Dr. Tanya Simuni. The remaining part of the conference discussed specific strategies and current protocols in development, including the SlowSPEED trial, P2P master, and PRISMS. Moreover, challenges were also highlighted, including the limitations of the current biological definitions, as well as the presence of co-pathologies and ethical concerns regarding interventions or testing of high-risk populations.
According to the organizers, the next steps for a prevention trial would include, “to identify the participants that you are interested in recruiting for the study. In a secondary prevention trial, we would identify individuals who are genetically or prodromally at risk for developing traditional motor PD. Although there are many candidate interventions including combinations for secondary prevention trials, initial prospects under investigation appropriately include low-risk therapies like aerobic exercise or repurposed agents, given that at-risk participants may require long-term therapy and may, regardless of their participation in the trial or not, never develop PD. Lastly, the primary outcome(s) of interest must be chosen. It may be clinical in nature, like a change in a parkinsonian motor, nonmotor, or patient-reported outcome measure (PROM) scale, or the development of traditional motor PD (i.e., phenoconversion). It may include a change in biomarkers, ranging from digital motor metrics via a wearable device, to an imaging signal pattern, to a biological fluid analyte or property.”
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