Abstract
Tremendous progress in our understanding of the pathophysiology and clinical manifestations of the prodromal phase of Parkinson disease (PD) offers a unique opportunity to start therapeutic interventions as early as possible to slow or even stop the progression to clinically manifest motor PD. A Parkinson's Prevention Conference, "Planning for Prevention of Parkinson's: A trial design symposium and workshop"was convened to discuss all issues that need to be addressed before the launch of the first PD prevention study. In this review, we summarize the major opportunities and challenges in designing prevention trials in PD, organized by the following critical trial design questions: Who (should be enrolled)? What (to test)? How (to measure prevention)? and the pivotal question, When during the prodromal disease (should we start these trials)? We outline the implications of these questions and their meaning for a responsible, sustainable, and fruitful further planning for prevention trials. Despite the great progress that has been made, it needs to be acknowledged that several queries remain to be carefully considered and addressed because prevention trials are being planned and become a reality.
| Original language | English (US) |
|---|---|
| Pages (from-to) | S76-S83 |
| Journal | Neurology |
| Volume | 99 |
| Issue number | 7 |
| DOIs | |
| State | Published - Aug 16 2022 |
Funding
The Article Processing Charge was funded by Massachusetts General Hospital. D. Berg reports no conflicts related to this paper. In the past 36 months, D. Berg reports the following, outside the submitted work: consultancies/advisory boards: Biogen, BIAL, UCB Pharma GmbH, and Zambon; honoraria: AbbVie, Biogen, BIAL, UCB Pharma GmbH, Zambon, and Desitin; grants: Deutsche Forschungsgemeinschaft (DFG), German Parkinson's Disease Association (dPV), BMBF, ParkinsonFonds Deutschland gGmbH, UCB Pharma GmbH, EU, Novartis Pharma GmbH, Lundbeck, and Damp Foundation. G.F. Crotty reports no conflicts of interest to report for this paper or in the past 36 months. J.L. Keavney reports no conflicts related to this paper. In the past 36 months, J.L. Keavney reports the following, outside the submitted work: consultancy with ESCAPE Bio. M.A. Schwarzschild reports no conflicts related to this paper. In the past 36 months, M.A. Schwarzschild reports the following, outside the submitted work: grants from the Michael J. Fox Foundation, NIH/NINDS, Farmer Family Foundation, Dept of Defense, and Burroughs Wellcome Trust; personal fees or travel for consulting or data monitoring committee service from Prevail Therapeutics, Denali Therapeutics, Eli Lilly, Parkinson Study Group (for nQ Medical, Chase Therapeutics, Partner Therapeutics, and Bial Therapeutics), Cure Parkinson's Trust, Corticobasal Solutions, Sutter Health/NIH/NIA, Parkinson's Foundation, and Michael J. Fox Foundation; and licensing fee distribution payments from MGH for adenosine A2A receptor knockout mice. T. Simuni reports no conflicts related to this paper. In the past 36 months, T. Simuni has served as a consultant for Acadia, Caraway Therapeutics, Critical Path for Parkinson's Consortium (CPP), Denali, General Electric (GE), Neuroderm, Sanofi, Sinopia, Sunovion, Roche, Takeda, MJFF, and Voyager. Dr. Simuni served on the advisory board for Acadia, Denali, General Electric (GE), Sunovian, and Roche. Dr. Simuni has served as a member of the scientific advisory board of Caraway Therapeutics, Neuroderm, and Sanofi. Dr. Simuni has received research funding from Biogen, Roche, Neuroderm, Sanofi, Sun Pharma, Amneal, Prevail, UCB, NINDS, MJFF, and Parkinson's Foundation. C. Tanner reports no conflicts related to this paper. In the past 36 months, C. Tanner reports the following, outside the submitted work: grants from the Michael J. Fox Foundation, NIH/NIA, NIH/NINDS, VA Merit, Dept of Defense, Parkinson's Foundation, Gateway LLC, Roche/Genentech, Parkinson Study Group, and Biogen Idec; personal fees for consulting or data monitoring committee service from Acorda, Adamas Therapeutics, Amneal, CNS Ratings, Gray Matter LLC, Northwestern University, Partners, Harvard University, Acadia, Neurocrine, Lundbeck, Cadent, and Kyowa Kirin; and nonfinancial support from Medtronic, Inc., Acadia, and Boston Scientific. Go to Neurology.org/N for full disclosures.
ASJC Scopus subject areas
- Clinical Neurology