The landmark PPMI study is amassing the most robust Parkinson’s dataset and biosample library in the history of Parkinson’s research, leading to scientific discovery and biomarker validation. PPMI is also a cornerstone for collaboration and a model for trial design.
In 2010, The Michael J. Fox Foundation and a handful of industry collaborators launched the first-ever large-scale clinical study exclusively focused on identifying and validating Parkinson’s disease biomarkers. While its mission toward objective biological measures of Parkinson’s has not changed, the impact of the Parkinson’s Progression Markers Initiative (PPMI) study is felt well beyond the borders of biomarker research.
Collecting a vast array of clinical and imaging data and biosamples from 1,400 volunteers at 33 clinical sites around the world over many years, PPMI has built the most robust dataset and biosample library in the history of Parkinson’s research. For example, the dataset includes whole exome and genome sequencing data, and fibroblasts and iPSCs are available.
Since its early days, the study has made its data available for download by any qualified researcher and taken applications for use of its biosamples.
PPMI continues to evolve — for example, adding new cohorts and data and sample collections, including brain tissue donation and wearable devices. PPMI’s industry collaborators — now up to 26 supporting the study through financial or in-kind donations and with intellectual expertise — have driven its growth and influence to this point and have a hand in its future.
Key Deliverables and Results
The impact of the PPMI study reaches across Parkinson’s research:
Source for Scientific Findings
Analysis of the PPMI dataset and biosamples has led to seminal research findings driving study focus and design. For example, people with Parkinson’s show significant, progressive loss of dopamine activity as measured by DaTscan in the first years after a diagnosis. And, predictive models and biological findings are helping to subtype the population.
Cornerstone for Validation and Collaboration
As PPMI makes its data available to the broad research community, many turn to this resource for discovery and validation of findings. PPMI data has been downloaded more than 2.8 million times.
In addition, the rigorous, standardized data and biosample collection across study sites established by PPMI have been adopted by the broader Parkinson’s field, enabling cross-study comparisons. The Accelerating Medicines Partnership Parkinson’s disease program (AMP PD) — launched in 2018 by the National Institutes of Health (NIH) with MJFF and five industry collaborators and managed by the Foundation for the NIH — is exploring molecular fingerprints in biofluids from Parkinson’s patients using samples collected through PPMI and other studies that modeled its methods.
Model for Trial Design
As more therapies enter and progress through clinical trials, companies are examining PPMI data to guide trial design. Adopting the same eligibility criteria, integrating DaTscan as an outcome measure and using data for predictive modeling to determine sample size are helping study sponsors design more efficient and effective disease-modifying trials. Additionally, PPMI’s success in recruiting and retaining volunteers for a long-term, intensive study has helped bolster confidence that drug trials will be able to do the same, thus aiding in investment decisions.
Industry collaborators are supporting PPMI through financial and in-kind contributions and are playing a lead role in providing feedback on study parameters through the Industry Scientific Advisory Board (ISAB). Their scientific thought leadership ensures that this dynamic study continues to meet the needs of industry to validate biomarkers of PD, inform trial design based on trends in industry, and de-risk the disease-modifying therapy landscape.
PPMI industry collaborators meet regularly by teleconference and once per year in person at the study’s annual meeting.
Read more about PPMI study design and findings and download study data through the PPMI website.
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