Accelerating Medicines Partnership - Parkinson's Disease

Combining data and resources to develop novel treatments for Parkinson’s disease

The Problem
Parkinson’s disease remains incurable due to a lack of data and resources and biomarker tools needed to assess a candidate treatment’s effectiveness during early-stage clinical trials.
The Solution
AMP PD will generate and combine data from thousands of PD patients across studies, to identify biomarker tools that will be informative to clinical trials of novel treatments.

Overview

The Accelerating Medicines Partnership for Parkinson’s disease (AMP PD) is a public-private partnership among NIH and multiple private sector organizations. Managed through the Foundation for the NIH, this partnership seeks to identify and validate biomarkers for diagnosis and disease subtyping as well as prognosis and monitoring progression that will provide new tools for use in clinical trials and speed up the development of novel therapies. 

AMP PD joins five other AMP programs, which are all coordinated by FNIH, that use cutting-edge scientific approaches to bring new medicines and supports to patients by enhancing validation of novel, clinically relevant therapeutic targets and biomarkers. Click here to learn more about AMP in general. To learn about AMP Type 2 Diabetes click here, AMP RA/SLE Project click here, AMP Alzheimer's Disease click here or AMP Schizophrenia click here.

Partners

Public-Sector Partners

  • National Institute of Neurological Disorders and Stroke (NINDS)
  • National Institute on Aging (NIA)
  • U.S. Food and Drug Administration (FDA)

Private-Sector Partners

  • Aligning Science Across Parkinson’s (ASAP) Initiative*
  • Celgene*
  • GlaxoSmithKline*
  • The Michael J. Fox Foundation for Parkinson’s Research (MJFF)*
  • Pfizer*
  • Sanofi*
  • Verily*

Provided financial or in-kind support for this program.

FNIH Contact

Eline Appelmans, M.D., MPH, BMedSci, Scientific Project Manager, Neuroscience, eappelmans@fnih.org

Parkinson's Disease

PD is a chronic and progressive neurological disease characterized by resting tremor, muscle rigidity, slowness of movement, impaired balance and a shuffling gait. Many people with PD also develop non-motor symptoms, such as behavioral changes and cognitive impairment. Although it is a complex neurodegenerative disorder, the two primary pathological hallmarks of PD are the death of dopamine neurons in a specific area of the brain called the substantia nigra, and buildup within neurons of a protein, alpha synuclein, into clusters called Lewy bodies. Because of the enhanced longevity of the worldwide population, PD represents a significant and ever-increasing threat to public health. In the United States alone, it is estimated that more than 1 million people live with PD and approximately 60,000 Americans are diagnosed with the disease each year. The current economic burden of PD is at least $14.4 billion per year and the prevalence of the disease will more than double by the year 2040.
 

Need for New Therapies

PD has been linked to several genetic, epigenetic and environmental factors; however, the exact causes of neuronal death and Lewy body formation remain unknown. Despite several hundreds of millions of dollars spent on research and development, no disease-modifying drugs have been approved for PD. Disease modifying treatments are those that can change the trajectory of disease progression and improve outcomes.

The data from ongoing  PD studies remains siloed behind independent access mechanisms, with no centralized harmonized dataset – limiting the ability to conduct the cross-study analyses required to develop a deeper mechanistic understanding of disease drivers that are needed for the success of novel therapies in clinical trials. 

AMP Approach

The success of clinical trials aimed at developing new treatments for PD hinges on identifying and validating biomarkers for application in phase II proof-of-concept trials. A wealth of information has been collected from individuals with PD through such programs as the NINDS’s Parkinson’s Disease Biomarkers Program (PDBP), the Michael J. Fox Foundation (MJFF)’s Parkinson’s disease Progression Biomarker Initiative (PPMI), the MJFF/NINDS BioFIND cohort and the Harvard Biomarkers Study (HBS). However, the prohibitive time, cost and resources required to perform a large-scale analysis of data from these programs have prevented individual researchers, companies and organizations from fully leveraging those datasets. Because it is a public-private partnership, AMP PD will provide the expertise and resources needed to determine which biomarkers show the greatest potential for predicting PD and the progression of the disease.

AMP PD Knowledge Portal

Industry partner Verily (Alphabet Inc.’s research organization dedicated to the study of life sciences) is has created a Cloud platform to house the data derived from biospecimen samples previously collected. These data and analyses are available to all AMP PD partner organizations and to the broader research community. The AMP PD researchers are collaborating to identify the most promising biomarkers for use in clinical trials for new treatments to affect the progression of PD.

The Introductory Webinar Getting Started with Accelerating Medicines for Parkinson's Disease (AMP PD) Knowledge Platform is now available for viewing on YouTube:

Governance

The Steering Committee for AMP PD consists of representatives from each of the research partner organizations. The Steering Committee operates under the direction of the overall AMP Executive Committee and is managed by the FNIH.

Support

To date, the FNIH has raised a total of $12 million in private-sector partner contributions over 5 years to fund AMP PD: $2 million/5 years each from Celgene, the Michael J. Fox Foundation, GSK, Pfizer and Sanofi, with an additional $2 million/5 years committed in-kind to develop the Knowledge Platform from Verily. NINDS has committed matching funds of $12 million/5 years, bringing the total funding for AMP PD to $24 million. Although this budget is adequate to fund the initial research plan designed by AMP PD partners, additional funds would be welcome in order to increase the number of patient samples and data types included in the biomarker analyses, and to enable application of newly emerging analytical platforms and techniques to the patient samples.

Results & Accomplishments

AMP PD Knowledge Portal and Platform houses harmonized multi-omic data from thousands of PD study participants and health controls.

Media

  • Developing therapies for Parkinson's disease. The Lancet Neurology (October 1, 2020). Editorial, Volume 19, Issue 10, 797. Read More
  •  Google Education:NIH program builds knowledge platform on Terra and Google Cloud to accelerate Parkinson’s Disease (PD) research Read more
  • FNIH Press Release (Nov.22, 2019): Accelerating Medicines Partnership launches data knowledge portal for Parkinson's disease Read more
  • Massive. NIH-industry project opens portals to target validation Nature Reviews Drug Discovery (February 27, 2019) Read more
  • NIH Press Release (Jan. 30, 2018): NIH Launches Partnership to Improve Success of Clinical Trials for Patients With Parkinson’s Disease Read more
  • Accelerating research for Parkinson's disease:The Lancet Neurology (April 1, 2018). Volume 17, Issue 4, 289. Read more

Scientific Publications

  • Accelerating Medicines Partnership: Parkinson's Disease. Genetic Resource. Movement Disorders. 07 May 2021. Hirotaka Iwaki MD,  Hampton L. Leonard MS,  Mary B. Makarious BS,  Matt Bookman MS,  Barry Landin BS,  David Vismer BS,  Bradford Casey PhD,  J. Raphael Gibbs PhD,  Dena G. Hernandez PhD,  Cornelis Blauwendraat PhD,  Daniel Vitale MS,  Yeajin Song MS,  Dinesh Kumar PhD,  Clifton L. Dalgard PhD,  Mahdiar Sadeghi MS,  Xianjun Dong PhD,  Leonie Misquitta PhD,  Sonja W. Scholz PhD,  Clemens R. Scherzer MD,  Mike A. Nalls PhD,  Shameek Biswas PhD,  Andrew B. Singleton PhD,  Uniformed Services University of the Health Sciences Associates  AMP PD Whole Genome Sequencing Working Group and  AMP PD consortium. Manuscript ID - MDS-20-1750.R1. https://doi.org/10.1101/2020.11.19.20235192