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Parkinson's Progression Markers Initiative (PPMI)

Trojanowski, J.Q.
American Association of Neuropathologists (AANP): Charleston, SC
Download Presentation: Trojanowksi-PPMI-AANP-6-21-2013-Final.pdf
Biomarkers for the early diagnosis of Parkinson Disease (PD) and to map PD progression as well as to monitor target engagement for disease modifying therapies would accelerate the pace of research to elucidate mechanism of PD and discover effective therapies. Despite progress to identify and validate PD biomarkers, no fully validated biomarker for PD is available while advances in molecular genetics, neurobiology and imaging technologies for PD have been remarkable. Hence, the lack of PD biomarkers is a significant roadblock for further studies of PD mechanisms and for discovery of disease modifying therapies. For these reasons, a major initiative to develop PD biomarkers is urgently needed, and this has lead to the launch of the Parkinson Progression Marker Initiative (PPMI). This is a remarkable public-private consortium to establish a collaborative effort of PD researchers with expertise in biomarker development, PD clinical study design and implementation, bioinformatics, statistics, and data management that is designed to identify and validate PD progression markers. PPMI is largely sponsored by the Michael J Fox foundation with substantial industry partnerships. PPMI is a five-year observational, international, multi-center study designed to identify PD progression biomarkers both to improve understanding of disease etiology and course and to provide crucial tools to enhance the likelihood of success of PD disease modifying therapeutic trials. The specific goals of PPMI are to: 1) Establish standardized protocols for acquisition, transfer and analysis of clinical, imaging and biospecimen data that can be used by the PD research community; 2) Investigate existing and identify novel clinical, imaging and chemical biomarkers that serve as PD diagnostic and progression markers that individually or in combination will rapidly demonstrate interval change in PD patients in comparison to healthy controls or in sub-sets of PD patients defined by baseline assessments, progression milestones and/or rate of clinical, imaging or biosample change; 3) Optimize bioassays and conduct preliminary verification studies on promising biological markers using stored biospecimens. PPMI was planned during a two-year period in a series of workshops with input from academic PD experts, Michael J Fox Foundation scientific staff, and government and industry partners. The study was launched in June 2010 and enrollment is nearly complete. This presentation provides an overview of the overall study design and key study features as well as the organizational structure of PPMI and some of the initial findings to date.