Help shape the first trials for the prevention of Parkinson’s disease

---

Over the past few years the science of conducting rigorous, rational clinical trials of candidate preventative therapies for Parkinson’s disease (PD) has gone from a dream to a reality. This trial science breakthrough relied on advances in defining populations at high risk for PD — based on prodromal features (e.g., hyposmia and REM sleep behavior disorder [RBD]) combined with pathogenic gene variants (e.g., of GBA and LRRK2) and accessible, early diagnostic biomarkers (of dopaminergic deficits and alpha-synuclein pathology). Several trials around the world — in Asia, Europe, and North America — have begun enrolling people with RBD, and others will soon start with recruitment centered on hyposmia or PD genes.

Motivated by this progress but also by building frustrations from decades of null results in prior ‘disease-modification’ trials enrolling people with already diagnosed PD, in 2021 we began convening a series of clinical trial design forums to envision, plan, and now compare the first PD prevention trials of pharmacological and lifestyle (e.g., exercise) interventions so that a wide range of stakeholders — at-risk advocates, biologists, clinical trialists, foundation and industry representatives, and regulators — could exchange ideas in a pre-competitive space to improve emerging trial designs and their prospects for success. Attendees have collectively addressed and workshopped the many remaining challenges in this nascent field. These range from ethical issues in recruiting generally healthy people to treat them to prevent diseases they may not develop for years (if ever even) without treament, to crucial methodological issues of how to best measure progression prior to a traditional diagnosis.

Planning for Prevention of Parkinson’s and Related Synucleinopathies: A Trial Design Forum, the 4th in this now biennial series, will be May 22-24, just before and nearby the World Parkinson’s Congress in Phoenix. Attendees from advocacy, academia, industry, government, philanthropy, and beyond are welcome to join in. A novel mentorship program has been designed for early-career investigators who present their work at conference poster session.

Late-Breaking abstracts may be submitted through March 6.

Find information about the conference, its program, sessions, speakers, registration, hotel booking, and late-breaking abstract submission:

Learn more

 

References:

Perspectives of People At-Risk on Parkinson's Prevention Research.
Keavney JL, Mathur S, Schroeder K, Merrell R, Castillo-Torres SA, Gao V, Crotty GF, Schwarzschild MA, Poma JM.J Parkinsons Dis. 2024;14(3):399-414. 

Designing the First Trials for Parkinson's Prevention.
Crotty GF, Ayer SJ, Schwarzschild MA.J Parkinsons Dis. 2024;14(s2):S381-S393.