The Foundation for the National Institutes of Health Announces Research Effort to Speed Parkinson’s Diagnosis and Enable Earlier Intervention

• The Accelerating Medicines Partnership® in Parkinson’s Disease and Related Disorders (AMP® PDRD) will expedite identification of biomarkers to better differentiate Parkinson’s from diseases with similar symptoms.
• Results could help avoid delays in diagnosis and lead to personalized treatments for those living with Parkinson’s.

NORTH BETHESDA, Md., July 17, 2024 / FNIH / —The Foundation for the National Institutes of Health (FNIH) announces a new public-private partnership that aims to better differentiate Parkinson’s disease from related neurodegenerative disorders, such as multiple system atrophy, Lewy body dementia, and progressive supranuclear palsy, to enable earlier diagnoses, more timely interventions, and better outcomes.

The Accelerating Medicines Partnership® in Parkinson’s Disease and Related Disorders (AMP® PDRD), managed by the FNIH, brings together the resources and expertise of the National Institute of Neurological Disorders and Stroke and the National Institute on Aging at the National Institutes of Health (NIH), the Food and Drug Administration (FDA), The Michael J. Fox Foundation for Parkinson’s Research, and stakeholders from the private sector, with a combined commitment of approximately $21 million.

“Although significant progress has been made in Parkinson’s research, there remains a need to deepen our understanding of how this disease begins and progresses over a long period of time,” said Julie Gerberding, MD, MPH, president and CEO of the FNIH. “By combining resources and expertise, this collaborative partnership promises to advance research more quickly and improve outcomes for individuals and families living with Parkinson’s and similar disorders.”

Due to increased life expectancy, Parkinson’s disease is a growing health challenge, affecting an estimated one million individuals in the U.S. and more than 8.5 million people worldwide. Available treatments can manage symptoms, but currently Parkinson’s disease has no cure.

The disease is characterized by tremors, muscle stiffness, slow movement, problems with balance and coordination, and a shuffling gait. People with Parkinson’s can also experience non-motor symptoms, such as changes in mood, behavior, and cognitive function. Some individuals develop a set of symptoms years or decades before motor signs occur, including a particular sleep disorder, constipation, and decreased sense of smell. Although the root causes of Parkinson’s remain unknown, risk factors include age, genetics, and exposure to environmental hazards.

Early diagnosis is crucial for initiating appropriate treatment for Parkinson’s disease. Doctors have traditionally relied on a patient’s medical history, neurological examinations, and imaging tests to rule out other conditions with similar symptoms. A definitive diagnosis can take several years after symptoms appear, and the misdiagnosis rate has been estimated at 10-20%. More recently, researchers developed a promising diagnostic test that can detect abnormal amounts of a protein called alpha-synuclein, a known biological characteristic of Parkinson’s disease, collected through either a spinal tap or skin biopsy.

AMP PDRD seeks to identify and validate additional biomarkers—particularly in more accessible tissues like blood or saliva—that can accurately differentiate Parkinson’s from related diseases, known as “Parkinsonism,” such as multiple system atrophy, Lewy body dementia, and progressive supranuclear palsy. New biomarkers to accurately diagnose Parkinson’s at an early stage can enable appropriate treatment years before major neurological disability.

Differentiating patients with Parkinson’s disease from patients with Parkinson-like disorders using validated biomarkers will also help to recruit the right participants in clinical trials for new drugs, thereby increasing the success rate of the trials. In addition, the project will generate and compare data from individuals at various stages of Parkinson’s to uncover insights into the onset and progression of the disease, leading to more precise and effective treatments.

“The expansion of AMP PDRD brings together scientists from industry, academia, and government to identify and validate biomarkers that can be used to detect whether an intervention has the desired effect when tested in people,” said Walter Koroshetz, MD, director, National Institute of Neurological Disorders and Stroke. “This will ultimately mean more quickly identifying which treatments are more likely to have beneficial effects for persons with Parkinson’s disease.”

AMP PDRD builds on an earlier project, the Accelerating Medicines Partnership in Parkinson’s Disease (AMP PD), which consolidated data from various studies and increased the number of bio-samples for research to help advance the discovery and development of disease-modifying treatments for Parkinson’s. A key result was establishing the AMP PD Knowledge Platform, which hosts genetic and other biological analysis data collected over time, enabling biomarker identification. AMP PDRD will expand the collected datasets to include a wider range of individuals suffering from Parkinson’s and related disorders, identify new biomarkers and targets for drug development, and improve data analysis tools for classifying disease stages and subtypes more precisely.

Todd Sherer, PhD, chief mission officer, The Michael J. Fox Foundation, said of the partnership, “Data sharing at this scale will accelerate the field’s progress by improving drug trials and fueling the clinical pipeline. The Michael J. Fox Foundation looks forward to the results of bringing so many knowledge bases together through this public-private consortium.”

The Accelerating Medicines Partnership in Parkinson’s Disease and Related Disorders is supported by the National Institute of Neurological Disorders and Stroke, the National Institute on Aging, the FDA, Aligning Science Across Parkinson’s, C2N Diagnostics, CurePSP, Denali, GSK, Sanofi, and The Michael J. Fox Foundation. An updated list of partners is available here. Read what the partners are saying here.

AMP PDRD underscores the FNIH’s commitment to tackling neurodegenerative disorders and follows the May launch of AMP Amyotrophic Lateral Sclerosis (ALS), an FNIH-managed public-private partnership targeting advancements in ALS diagnostics and treatments.

About the Accelerating Medicines Partnership

Launched in 2014 and managed by the FNIH, the Accelerating Medicines Partnership® (AMP®) program brings together the National Institutes of Health, U.S. Food and Drug Administration, biomedical and life science companies, nonprofits, patient-focused groups, and other organizations to transform the current model for developing new diagnostics and treatments. Using cutting-edge scientific approaches and broad sharing of research data, all AMPs seek to improve understanding of disease pathways, facilitate better selection of targets for drug development, and streamline processes for bringing new treatments to patients. AMP PDRD is the latest initiative in a portfolio of AMP projects expediting discovery around amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, schizophrenia, common metabolic diseases, heart failure, autoimmune and immune-mediated diseases, and rare diseases. To learn more about AMP, visit fnih.org/AMP.

About the Foundation for the National Institutes of Health

The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH) with their counterparts in life sciences companies, academia, patient organizations, foundations, and regulatory agencies (including the Food and Drug Administration and the European Medicines Agency). Through team science, the FNIH solves complex health challenges and accelerates breakthroughs for patients, regardless of who they are or what health threats they face. The FNIH contributes to the development of new therapies, diagnostics, and potential cures; advances global health and equity in care; and celebrates and helps train the next generations of scientists. Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org.