Objective: The overall goal of PPMI is to identify, test and validate biomarkers of PD to accelerate therapeutic development throughout the PD continuum.

Background: PPMI has developed a robust clinical, imaging, and genetic dataset and biosamples from longitudinally followed participants with sporadic PD, PD with genetic variants, healthy subjects, and individuals at risk due to RBD, olfactory dysfunction and/or PD genetic variants. Data are collected and analyzed according to standardized protocols. All data collected is open source, available at www.ppmi-info.org.

Method: PPMI is enrolling at 52 sites worldwide including participants with clinical diagnosis of PD, with biomarkers/early clinical markers for PD and Healthy Controls. PPMI developed a strategy using a staged risk paradigm to identity and enroll participants with biomarkers and/or early clinical markers of PD. These participants are identified through online platforms (PPMI Online, Healthy Brain Ageing (HeBA)) and a broad outreach via smell test direct (ST Direct), inviting participants to test olfaction to assess PD risk. Participants with olfactory dysfunction are tested with dopamine transporter imaging to determine eligibility.  Eligible participants are enrolled in clinic for longitudinal follow-up including clinical, imaging, and biosample collection (blood, CSF, skin biopsy) to assess genetics and biologic measures of synuclein, lysosomal function, and analytes related to neurodegeneration.

Results: PPMI has enrolled 1693 participants (798 clinical PD, 721 biomarkers/clinical markers for PD, 174 healthy subjects).  More than 100,000 participants have enrolled in online platforms (PPMI online 35000, ST Direct 70000, HeBA 10000). Data has confirmed that olfaction is a sensitive predictor of DAT deficit.  Recent data shows that synuclein aggregation assays can detect synuclein in RBD and olfactory impaired participants prior to the onset of PD clinical symptoms providing an additional biomarker to identify early PD pathology.

Conclusion: PPMI has successfully established a novel, wide-ranging enrollment strategy across the PD continuum including participants with both biomarker/clinical markers and clinical manifestations of PD.  Biomarker studies in PPMI participants focused on synuclein and dopamine dysfunction have provided a framework to define PD biology and advance PD therapeutics.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/parkinsons-progression-markers-initiative-ppmi-defining-pd-biology-to-accelerate-pd-therapeutics/