Objective: The Parkinson Progression Marker Initiative (PPMI) is expanding its cohorts and exploring additional biomarkers of PD progression to test hypotheses of the underlying molecular pathobiology of PD,  enable modeling of PD progression to identify clinical and/or biologic data driven PD progression sub-sets, and to inform studies testing PD therapeutics.

Background: The PPMI study was launched in 2010 and has made substantial progress to establish a robust clinical and biomarker data set and biosample repository that has contributed to the study design of numerous planned and ongoing clinical trials in PD. A major goal of the expanded PPMI is to identify biomarker signatures across the PD continuum from prodromal to moderate PD.  The overall goal of PPMI is to identify markers of disease progression for use in clinical trials of therapies to reduce progression of PD disability.

Method: The expanded PPMI includes PPMI Clinical, PPMI Online and Remote, and PPMI Digital Applications.  In  PPMI clinical, approximately 4000 PD, healthy and prodromal participants will undergo comprehensive, longitudinal clinical and biomarker phenotyping for at least five years.  PPMI online and remote will evaluate  participants who may be at risk for PD  via a staged risk paradigm starting with  PPMI-online, a custom-built online data platform, and followed by targeted remote assessments including olfactory testing,  Those eligible based on their remote testing would then be further evaluated with DAT imaging to determine eligibility for prodromal PPMI Clinical.

Results: Key findings from PPMI include 5-year longitudinal MDS-UPDRS follow-up in an early PD cohort,  longitudinal DAT imaging demonstrating robust reduction of 28% during 4-year follow-up in PD patients, and reduction in CSF synuclein, Tau, pTau of 15-20% observed at diagnosis and during 3-year longitudinal follow up in PD vs healthy participants. PPMI data and samples are available to the PD community via the PPMI website (www.ppmi-info.org).

Conclusion: PPMI has established a  robust research infrastructure and a widely used PD data and sample resource.  The expanded PPMI will enable PPMI to comprehensively assess clinical and biomarkers signatures throughout the PD continuum. This strategy has the potential to establish a framework for identifying  a prodromal PD cohort and meaningful clinical and biomarker outcomes that could accelerate clinical trials of both manifest and prodromal PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/parkinson-progression-marker-initiative-new-science-new-cohorts/