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Longitudinal PD Biomarker Studies: DeNoPa and PPMI

Mollenhauer, B
AD/PD: Nice, France
Download Presentation: Mollenhauer-AD.PD-2015.pdf
OBJECTIVES: To demonstrate first longitudinal data on one single- [De No Parkinson?s disease (PD): DeNoPa] and one multicenter biomarker study (Parkinson Progression Marker Initiative of the Michael J Fox Foundation; PPMI) for progression biomarkers as outcome measures for clinical trials

METHODS: clinical measures (motor scales, non-motor symptoms, cognitive tests, sleep),volumetric imaging [magnetic resonance imaging (MRI)] and blood/cerebrospinal fluid (CSF) biomarkers have been investigated in two longitudinal cohorts on de novo PD and healthy controls.

RESULTS: 159 patients and 110 healthy controls have been recruited in the DeNoPa study and followed after 24 months. A multimodal panel of clinical, functional and imaging measures significantly reflect the progression of the disease in DeNoPa.While non-motor symptoms progress (MDS-UPDRS 1 and Scopa AUT), sleep deteriorates (ESS) and volumetric imaging of grey-matter and hippocampal area decrease significantly, the CSF parameters remain relatively stable (?-synuclein) or increase slighlty (?-amyloid 1-42, total tau protein, neurofilaments) from baseline to the first follow-up period in comparison of patients and controls. This propoed progression panel needs validation by continuing follow-up and in independent, larger cohorts, such as PPMI. For PPMI 24 sites in the USA and Europe recruited 232 de novo PD patients and 196 healthy controls. Follow-up assessments including clinical data, MRI, dopamine transporter imaging, CSF and blood sampling are done every 6 months.

CONCLUSION: A multimodal panel of progression marker can serve as outcome measure for clinical trials in de novo PD. More accurate biomarkers in biological fluids for the progression of PD need to be established.