Objective: PPMI is a longitudinal, observational study enrolling PD participants, individuals at risk for PD, and healthy controls (HCs). The study investigates clinical, imaging, genetic and biofluid biomarkers to elucidate the course of PD from start of neurodegeneration through clinical PD to accelerate therapeutic development.

Background: Since 2010 PPMI has evolved into a broad international study demonstrating the natural history of PD clinical and imaging progression. PPMI has acquired comprehensive longitudinal data and biosamples including CSF. Recent data has utilized the unique PPMI resource to validate the α-synuclein seed amplification assay (asyn SAA) in CSF. PPMI data is open source, available at www.ppmi-info.org

Method: The PPMI Program enrolls and follows densely phenotyped populations (n=3000) at 50 clinical sites worldwide. Participants include a PD newly diagnosed untreated cohort, a prodromal cohort focused on RBD and hyposmia and HCs. Participants are assessed annually for at least 5 years using clinical (motor and non-motor) scales (e.g. MDS-UPDRS, MoCA), Patient Reported Outcomes (PROs), quantitative imaging (DAT, SBR, MRI midbrain melanin), and biologic measures of synuclein, lysosomal function, and analytes related to neurodegeneration. PPMI also has enrolled >100,000 participants in remote assessment strategies, which have been aggregated under myPPMI, a web portal that provides opportunities for acquisition of data and return of personalized data to study participants.

Results: Recent data demonstrate that >90% of PD participants and approx. 50-70% of RBD and hyposmic participants are asyn SAA+. These data coupled with DAT imaging and olfactory function results have led to a biomarker driven definition for PD and the introduction of the concept of Neuronal Synuclein Disease (NSD) encompassing PD, dementia with Lewy Bodies and RBD. PPMI continues to follow these participants and plans to enroll individuals at the earliest stages of neurodegeneration to further elucidate the progression of NSD.

Conclusion: PPMI has successfully enrolled and followed study participants throughout the course of disease and continues to maintain open-source clinical data and biomarker repository that informs our NSD definition and understanding of PD progression. PPMI data will continue to expand and evolve this definition with additional comprehensive longitudinal data acquired at all stages of disease.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/parkinsons-progression-markers-initiative-ppmi-investigating-biomarker-driven-pd-progression/