Accelerating Medicines Partnership - Parkinson's Disease

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The Accelerating Medicines Partnership (AMP) is a public-private partnership between the NIH, FNIH, multiple biopharmaceutical companies and not-for-profit organizations. Managed through the FNIH, AMP aims to identify and validate the most promising biological targets for therapeutics. In 2016, the AMP Executive Committee approved the planning of an AMP effort to confront the challenges presented by Parkinson’s disease (PD). This complements current efforts in the areas of Alzheimer’s disease, type 2 diabetes and the autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus). A critical component of this partnership is that all members have agreed to make the AMP Parkinson’s disease (AMP PD) data and analyses publicly available to the broad biomedical community.

Parkinson’s Disease

PD is a chronic and progressive neurological disease that is marked by resting tremor, muscle 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. Though 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 the buildup within neurons of a protein, α-synuclein, into clusters called Lewy bodies. Due to 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 each year. The economic burden of PD is at least $14.4 billion a year and the prevalence of the disease will more than double by the year 2040.

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 several hundreds of millions of dollars spent on 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.

AMP Approach

The success of clinical trials aimed at developing new treatments for PD hinges on identifying and validating biomarkers for application in phase 2 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 resources, time and cost required to perform a large-scale analysis of that data has been prohibitive for individual researchers, companies and organizations.

Through its public-private partnership, AMP PD will provide the expertise and support 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 creating a platform to house the data from cerebrospinal fluid, RNA, plasma and DNA samples previously collected from more than 3,000 PD cases and more than 1,700 healthy controls. These data and analyses will be made available across the entire partnership and to the broader research community. Once the Knowledge Portal is created and the existing PD data incorporated, the AMP PD researchers will collaborate to identify the most promising biomarkers for use in clinical trials for new treatments to affect the progression of PD.

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 organized by the FNIH.

Support

To date, the FNIH has raised a total of $12 million over 5 years in private sector partner contributions 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. While this is adequate to fund the initial research plan identified by the partners, additional funds would be welcome in order to increase the number of patient samples and data types included in the biomarker analyses, as well as enabling the application of newly emerging analytical platforms and techniques to the samples, expanding the program’s reach and ability to add to our knowledge of Parkinson’s disease.

Current Funding Opportunities:

AMP PD Request for Proposal 1

Instructions for Formatting:

Proposals should be limited to 20 pages (not including key proposed team members curricula vitae, limited to 4 pages each in standard NIH format, and letters of support). Please use 12 point font, single-spaced formatting with half-inch margins.

Results & Accomplishments

Media

Massive NIH-industry project opens portals to target validation. Nature Reviews Drug Discovery (February 27, 2019)

NIH Press Release (Jan. 30, 2018): NIH Launches Partnership to Improve Success of Clinical Trials for Patients With Parkinson’s Disease

Accelerating research for Parkinson's disease.The Lancet Neurology, Volume 17, Issue 4, 289. 

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:
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

Rosa Canet-Avilés, Ph.D., Director, Neuroscience, Research Partnerships rcanet-aviles@fnih.org