Accelerating Medicines Partnership® Program – Parkinson's Disease

Combining data and resources to aid discovery of 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® (AMP®) Program Parkinson’s disease (AMP PD) is a public-private partnership among NIH and private sector organizations. Managed by the Foundation for the NIH, this partnership seeks to identify and validate biomarkers for diagnosis, disease subtyping, prognosis and monitoring progression to provide new tools for use in clinical trials and speed up development of novel therapies. 

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 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 PD will more than double by the year 2040.

PD has been linked to several genetic, epigenetic and environmental factors; however, exact causes of neuronal death and Lewy body formation remain unknown. Despite 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.

Data from current PD studies remains siloed behind independent access mechanisms, with no centralized harmonized dataset – limiting the ability to conduct cross-study analyses required to develop deeper mechanistic understanding of disease drivers to be targeted by novel therapies in trials.

Success of clinical trials aimed at developing new treatments for PD hinges on identifying and validating biomarkers for application in clinical 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 these datasets. As a public-private partnership, AMP PD will provide expertise and resources needed to determine which biomarkers show the greatest potential for predicting PD and the progression of the disease.

Read more about AMP PD on the National Institute of Neurological Disorders and Stroke’s website.

Goals

  • Standardize data collection for biomarkers in multiple cohorts.
  • Conduct standardized assays on thousands of existing biosamples, incorporating existing clinical, imaging, genetic data.
  • Pursue additional large-scale biomarker discovery with transcriptomics, epigenomics, whole genome sequencing, metabolomics, and proteomics.
  • 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.

Results and Accomplishments

Industry partner Verily (Alphabet Inc.’s research organization dedicated to the study of life sciences) has created the AMP PD Knowledge Portal and Platform, a cloud-based platform that houses harmonized multi-omic data from thousands of PD study participants and healthy controls, derived from biospecimen samples previously collected. These data and analyses are available to all AMP PD partner organizations and to the broader research community. 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 the Accelerating Medicines Partnership program for Parkinson's Disease (AMP PD) Knowledge Platform” is now available for viewing on YouTube: Part 1, Part 2a, Part 2b, Part 3a, Part 3b, and Part 4.

Media

Google Education: NIH program builds knowledge platform on Terra and Google Cloud to accelerate Parkinson’s Disease (PD) research.

FNIH Press Release (November 22, 2019): Accelerating Medicines Partnership launches data knowledge portal for Parkinson's disease.

NIH Press Release (January 30, 2018): NIH launches partnership to improve success of clinical trials for patients with Parkinson’s disease.

Support

For more information about supporting or becoming a partner in this project, please contact Heidi Blythe, Director, Strategic Alliances and Advancement, hblythe@fnih.org.

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.