Objective: The goal of the Parkinson Progression Marker Initiative (PPMI) is to identify clinical, imaging and biospecimen biomarkers of PD progression to provide tools for studies of disease modifying therapeutics.

Background: Lack of progression biomarkers has been a major roadblock to studies investigating potential disease modifying PD drugs.

Methods: PPMI has enrolled 423 PD, 196 healthy subjects, 64 SWEDD, 65 prodromal PD (39 RBD and 26 hyposmic) and is enrolling 600 PD subjects and unaffected carriers with LRRK2, GBA, or synuclein mutations. All subjects will be evaluated longitudinally with MDS-UPDRS, non-motor cognitive and behavioral assessments, DAT imaging, biofluid sampling including blood and CSF at 6-12 month intervals. Access to PPMI data and biospecimen is available at www.ppmi-info.org.

Results: All PPMI PD, healthy subjects and SWEDD subjects have reached their 24-month assessment. MDS-UPDRS for all PD subjects (including those treated assessed in defined off) was 32.4 (13.1), 39.6 (16.2), 43.4 (17.0) at baseline, 12 months and 24 months. Approximately 60% of subjects required medication by 12 months and 80% by 24 months. Dopamine transporter contralateral putamen binding ratio was 0.69 (0.3), 0.61 (0.2), 0.56 (0.2) at baseline, 12 months and 24 months. Of the 64 SWEDD subjects at baseline 5 subjects showed DAT deficit at 24 months, 4 of 5 had quantitative DAT deficit at baseline. Prodromal PD subjects identified by RBD (with abnormal polysomnagram) plus DAT deficit (n=39) showed baseline MDS-UPDRS 14.0 (7.6) total, 4.5 (3.8) motor, MoCa 25.5 (4.1), GDS 2.8 (2.6), and Scopa-Aut 14.9 (8.2).

Conclusions: Longitudinal PPMI data acquired during 24 months follow-up demonstrates progression of UPDRS and DAT imaging. These data combined with genetic, non-motor and blood and CSF analytes can establish PD progression subsets and provide sample size estimates to inform PD study design for therapeutics designed to modify disease progression Finally longitudinal prodromal PD subjects will be followed to investigate the biomarker signature of phenoconversion. PPMI is sponsored by the Michael J. Fox Foundation for Parkinson’s Research (MJFF) and is co-funded by MJFF, Abbvie, Avid Radiopharmaceuticals, Biogen, Bristol-Myers Squibb, Biolegend, Eli Lilly & Co., F. Hoffman-La Roche, Ltd., GE Healthcare, Genentech, GlaxoSmithKline, Lundbeck, Merck, MesoScale, Piramal, Pfizer, Servier and UCB.

« Back to 2016 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/the-parkinson-progression-marker-initiative-ppmi-developing-a-comprehensive-longitudinal-biomarker-dataset/