Path 2 Parkinson's Prevention with Drs. Simuni and Wendelberger

In this episode of "In the Interim…", Dr. Scott Berry is joined by Dr. Tanya Simuni, Arthur C. Nielsen Jr. Professor of Neurology and Director of the Parkinson’s Disease and Movement Disorders Center at Northwestern University, and Dr. Barbara Wendelberger, Senior Statistical Scientist at Berry Consultants. The conversation focuses on the Path to Prevention (P2P) platform trial—an international, multi-arm prevention study in Parkinson’s disease targeting participants defined by biological markers, specifically alpha-synuclein pathology, prior to clinical diagnosis. The discussion covers the PPMI cohort, trial operational and statistical structure, the rationale behind biomarker-driven inclusion, and the use of Bayesian platform trial design.

Key Highlights:

• Parkinson’s disease pathobiology and risk: genotype-phenotype variability, multi-system involvement, and the central roles of age, environment, and genetics.
• Michael J. Fox Foundation’s PPMI cohort: 4,000+ participants, prospective longitudinal biomarker and clinical data, high participant retention, enabling study of early Parkinson’s.
• P2P platform structure: multi-arm design, two-stage randomization with shared placebo group, integration of non-randomized PPMI cohort in Bayesian analysis for improved inference.
• Inclusion criteria: prodromal population biologically defined by CSF alpha-synuclein seed amplification and dopaminergic imaging (DAT-SPECT), highlighting regulatory nuances.
• Dual primary endpoints: biomarker (DAT-SPECT) and clinical (MDS-UPDRS Part III), 24-36 months follow-up.
• Commitment to public data sharing in line with the Michael J. Fox Foundation’s open science philosophy.

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