This repository is under review for potential modification in compliance with Administration directives.
The AMP PD Knowledge Portal was developed to host and share resources related to Parkinson’s disease research and remains fully operational. We continue to maintain and accept Parkinson’s disease and related disorders data and resources throughout this review process.

Overview: About AMP PDRD and AMP PD

About the Accelerating Medicines Partnership® (AMP®) Program

About AMP-PD

The Accelerating Medicines Partnership® (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), multiple biopharmaceutical and life sciences companies, and non-profit organizations.

Managed through the Foundation for the NIH (FNIH), AMP aims to identify and validate the most promising biological targets for therapeutics. Disease areas covered by AMP at the launch of this site include Alzheimer’s disease, type 2 diabetes, the autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus) and Parkinson’s disease. Additional disease areas are being evaluated for addition to AMP.
 

AMP PDRD builds upon the infrastructure established during its first phase, AMP Parkinson’s Disease (AMP PD), which established a centralized data portal to enhance the broad sharing of harmonized data from diverse natural history and clinical trial studies to advance the discovery and development of therapeutics for people living with Parkinson’s disease.  This new partnership for Parkinson’s Disease and Related Disorders (AMP PDRD) extends beyond the first phase of AMP PD by enabling ways to better differentiate PD from related neurodegenerative disorders such as multiple system atrophy, Lewy body dementia, and progressive supranuclear palsy. AMP PDRD will employ a precision medicine-based, data-driven approach to identify and validate biomarkers distinguishing Parkinson’s disease from related diseases, including other α-synucleinopathies and atypical parkinsonisms. The goal of this program is to enable earlier diagnoses, more timely interventions, and better outcomes for people living with these disorders. What/Goals: Identify and validate proof of mechanism biomarkers tied to Parkinson’s Disease and other -synucleinopathies through 3 aims:

Target Biomarker Discovery and Validation

Identify and validate a panel of fit-for-purpose biomarker candidates for PD and Related Disorders which may inform patient stratification, monitoring of disease progression, enabling clinical trial design, and the potential to accelerate the development of meaningful therapies. 

Longitudinal Multi-omic Molecular Profiling

Build upon the current data generated by AMP PD with additional generation of harmonized multi-omic datasets including WGS, longitudinal RNA-Seq, longitudinal proteomics, scRNA-Seq, and others from postmortem tissue and clinical samples of interest on other α -synucleinopathy and atypical parkinsonism cohorts. 

Multi-scale Analyses Framework

Support data use and analysis which can leverage machine learning approaches to develop algorithms for prediction, prognosis, and progression of PD and related disorders. 

Why: Problem Statement for Parkinson’s Disease

Parkinson’s disease (PD) is a chronic and progressive neurological disease that is marked by tremors in the resting muscles, rigidity, slowness of movement, impaired balance and a shuffling gait. In addition, many people with PD develop non-motor symptoms such as behavioral changes and cognitive impairment. 

Because the worldwide population is living longer, PD represents a significant and increasing threat to public health. In the United States alone it has been estimated that up to 1 million people may have PD. That number is expected to increase in the coming years, and the current annual cost of treatment for PD in the United States has been estimated at $14.4 billion. 

Though it is a complex neurodegenerative disorder, the two primary hallmarks of PD are the progressive loss of dopamine-containing neurons in a specific area of the brain called the substantia nigra and the buildup within neurons of a protein, α-synuclein, into clusters called Lewy bodies.

Need for New Therapies

The cause of PD has been linked to several genetic, epigenetic and environmental factors; however, the exact causes of neuronal death and Lewy body formation are not known. 

Despite large investments in research and development, no disease-modifying drugs have been approved for PD. It has become clear that a breakthrough in this area will require strong and transformative public-private partnerships that can produce bold discoveries.

How: AMP Governance

AMP is a public-private partnership managed by the FNIH. NIH and industry partners share expertise and resources in an integrated governance structure that enables the best-informed contributions to science from all participants. Each AMP disease area has a steering committee comprising representatives from the administering NIH Institute, the FDA, industry, and nonprofit foundations. The disease-specific steering committees are managed by the FNIH under the direction of the AMP Executive Committee, which includes leaders from NIH, FDA, participating industry partners, and nonprofit foundations that include patient advocacy organizations. The AMP PDRD Steering Committee convenes monthly to discuss project plans and review ongoing progress and milestones. NINDS program staff members provide scientific and administrative direction and oversee the cooperative grants derived from the consortia. The AMP PDRD program has several working groups that bring together subject matter experts from academia and industry. Face-to-face meetings organized by FNIH provide an additional venue for communication and coordination.

The Accelerating Medicine Partnership in Parkinson’s Disease (AMP PD)

In 2018, through FNIH, an AMP partnership between the National Institute of Neurological Disorders and Stroke (NINDS), National Institute on Aging (NIA), the Food and Drug Administration (FDA), GSK, Pfizer, Sanofi, Bristol-Myers Squibb, Verily and the Michael J. Fox Foundation for Parkinson's Research (MJFF) was launched for Parkinson’s disease (PD).  Aligning Science Across Parkinson's (ASAP) initiative joined the partnership in 2019 and AbbVie joined the partnership in 2022 to expand project goals.

The research plan proposed for the AMP program in PD encompasses a deep molecular characterization and longitudinal clinical profiling of PD patient data and biosamples with the goal of identifying and validating diagnostic, prognostic and/or disease progression biomarkers for PD. 

A critical component of this partnership is broad sharing of the AMP PD data and analyses with the biomedical community to advance research in PD.  AMP PD utilizes well characterized cohorts with existing biosamples and clinical data that were collected under comparable protocols and using common data elements.

The Release 1.0 cohorts include the MJFF and NINDS BioFIND study, Harvard Biomarkers Study (HBS), the NINDS Parkinson's Disease Biomarkers Program (PDBP), and MJFF Parkinson’s Progression Marker Initiative (PPMI). Release 2.0 adds the following cohorts; NIA International Lewy Body Dementia Genetics Consortium Genome Sequencing in Lewy body dementia case-control cohort (LBD), the MJFF LRRK2 Cohort Consortium (LCC), and the NINDS Study of Isradipine as a Disease Modifying Agent in Subjects With Early Parkinson Disease, Phase 3 (STEADY-PD3). Release 2.5 adds the MJFF and NINDS Study of Urate Elevation in Parkinson’s Disease, Phase 3 (SURE-PD3).  Release 3.0 adds the Global Parkinson’s Genetics Program (GP2). Release 3.0 also adds additional targeted proteomics from PPMI and PDBP as well as new bulk exRNA pilot sample data, new WGS single sample data, and a new global inventory table. Release 4.0 adds Postmortem Brain Sequencing, Untargeted Proteomics Data, and normalized Targeted Proteomics Data.

Thank you to all of the research participants who have contributed data through BioFIND, GP2, HBS, LBD, LCC, PDBP, PPMI, STEADY-PD3, and SURE-PD3 and to all of the study teams who have collected and managed these data. AMP PD appreciates your contributions, which are fundamental to AMP PD resource development. 

What: Goals for AMP PD

AMP PD aims to identify and validate diagnostic, prognostic, and progression biomarkers. This will improve clinical trial design and contribute to the identification of new pathways for therapeutic developments.

The goals of AMP PD are: 

  • To standardize data collection for biomarkers in multiple cohorts
  • To conduct standardized assays on thousands of existing biosamples, incorporating existing clinical, imaging, genetic data
  • To pursue additional large-scale biomarker discovery with transcriptomics, epigenomics, whole genome sequencing, metabolomics, and proteomics
  • To dissect new targets and disease subtypes; track and predict disease progression; identify biomarkers of Parkinson’s progression and assess their potential as targets for therapies

AMP PD & AMP PDRD Consortium

GovernmentAMP Partners

Food and Drug Administration (FDA)

National Institute of Neurological Disorders and Stroke (NINDS)

National Institute on Aging (NIA)

Industry

Bristol-Myers Squibb

GSK

Pfizer

Sanofi

Verily

AbbVie

Denali

C2N Diagnostics

Labcorp

Nonprofit

Aligning Science Across Parkinson's (ASAP) Initiative

The Michael J. Fox Foundation (MJFF)

Data

MJFF and NINDS BioFIND Study

NIA International Lewy Body Dementia Genetics Consortium Genome Sequencing in Lewy body dementia case-control cohort (LBD)

MJFF LRRK2 Cohort Consortium (LCC)

Brigham and Women's Hospital/MGH Harvard Biomarkers Study

NINDS Parkinson's Disease Biomarkers Program

MJFF Parkinson’s Progression Markers Initiative

NINDS Study of Isradipine as a Disease Modifying Agent in Subjects With Early Parkinson Disease, Phase 3 (STEADY-PD3).

The MJFF and NINDS Study of Urate Elevation in Parkinson’s Disease, Phase 3 (SURE-PD3)

Global Parkinson's Genetics Program (GP2)

Managing

Foundation for the NIH (FNIH)

The FNIH, managing partner for AMP PD, is actively recruiting new industry and nonprofit partners. Inquiries about how your organization can join the partnership should be submitted to Tyler Fortuna, Neuroscience Research Partnerships, at tfortuna@fnih.org.