Opening the doors to Understanding Parkinson's Disease
I am incredibly pleased to announce the recent launch of the Accelerating Medicines Partnership (AMP) Parkinson’s Disease (PD) Knowledge Portal. NIH has joined forces with public, private, and non-profit partners to provide a platform that will lower barriers to identifying and validating biomarkers that could be used in PD diagnosis, prognosis, mapping disease progression, and finding new therapeutic targets.
While caring for patients with PD, I and other neurologists have been impressed by the power of specific drugs and deep brain stimulation to treat the debilitating motor symptoms of PD. Unfortunately, these interventions have frustrating limitations: they do not slow or prevent the progression of PD, and as the disease continues to progress, their effectiveness declines. Multiple clinical trials have failed to identify a therapy that could stem the progressive neurodegeneration. A major stumbling block is the lack of biomarkers – e.g., blood or spinal fluid tests, brain scans, or other means to measure disease activity. In other conditions like multiple sclerosis, stroke, Huntington’s disease, and Alzheimer’s disease, early stage clinical trials are looking at the effects of treatments on such biomarkers. Promising outcomes in these early studies can increase the chances of success in in later stage testing through larger, longer, and more expensive clinical trials.
However, we in the research and clinical community lack specific tools to help diagnose PD, track its progression, and predict its outcome, and identifying biomarkers of PD activity could be a game-changer. From autopsy studies, we know that PD begins decades before classic symptoms appear. The search is on for biomarkers that predict PD and for effective disease-modifying treatments that can be started early, before the disease causes tremor, slowness of movement, balance trouble, and other symptoms. Coalescing efforts and pooling knowledge across the PD research and clinical communities would provide a unique opportunity to leverage resources toward goals to treat PD early and prevent disability, and ultimately to cure this disease.
Through AMP PD, and together with our private partners and grantees, we have built a data portal that brings clinical, genomic, and transcriptomic data on thousands of research participants under one “roof.” AMP PD is a platform that researchers can use to access and study these complex longitudinal clinical and -omics data from over 4000 participants, including both individuals with PD and healthy controls. This effort is part of the broader Accelerating Medicines Partnership (AMP) program, and AMP PD partners include the NIH, the Food and Drug Administration (FDA), GSK, Pfizer, Sanofi, Celgene, Verily and the Michael J. Fox Foundation (MJFF), all managed by the Foundation for NIH (FNIH). AMP PD has harmonized data sets from the MJFF/NINDS-sponsored BioFIND study, Harvard Biomarkers Study (HBS), the NINDS Parkinson's Disease Biomarkers Program (PDBP), and MJFF Parkinson’s Progression Marker Initiative (PPMI). This harmonization has ensured accurate data importing, consistent clinical descriptions, and intensive quality control. In addition to data currently available, we anticipate that AMP PD will expand to include additional clinical, proteomics, and metabolomics data.
The portal itself is a unique tool managed through a cloud platform that stores the data and allows analysis within the cloud. Instead of bringing the data to individual scientists, this system encourages scientists to come to the data, thereby reducing barriers such as computing power and siloed analysis. We encourage anyone interested in accessing the data to first complete the AMP PD Access Form which can be found on the AMP PD how-to website. When access is approved, the researcher can explore and analyze the data on their own through the cloud platform. No matter your background, becoming familiar with the user interface will help you get the most out of the portal resources. To orient researchers, NINDS and the AMP PD partners are hosting a webinar on December 5 where representatives will be able to answer your questions and teach you how the system works. We will archive the webinar and provide further tools to ensure that these data are as accessible as possible to all researchers.
We hope that providing a platform for researchers to easily share and analyze PD data will accelerate the identification and validation of PD biomarkers. These biomarkers will aid in determining who has PD, at what disease stage, and how it will progress. In a disease as variable as PD, these are necessary components to achieving the ultimate goal of accelerating PD therapy development. I am deeply grateful to all of the participants in the clinical studies that have provided data to AMP PD, the researchers who have contributed their time and effort to harmonize the data, and to many more who have worked on building AMP PD to where it is today. The AMP PD doors are open, and I encourage you to roll up your sleeves and start analyzing!
