So much Parkinson’s-related biomarker information has been collected under various research projects that sorting through it for additional clues to the disease poses a challenge to scientists.
The scope of the information makes a collaborative research effort paramount — and a U.S. government-sponsored initiative is meeting that need, according to an editorial in the journal The Lancet Neurology. The initiative is also focusing on therapy development.
It praised the Accelerating Medicines Partnership on Parkinson’s disease as the most ambitious effort so far to find a cure for Parkinson’s.
Led by the Foundation for the National Institutes of Health (NIH), the collaboration includes the US Food and Drug Administration (FDA), five biopharmaceutical and life science companies, and one non-profit foundation.
The aim of the partnership, formed on Jan. 30, 2018, is to discover new therapies for the disease plus new biomarkers that can predict its progression and whether treatments are working.
Scientists have collected a lot of Parkinson’s-related biosamples under programs such as the US National Institute of Neurological Disorders and Stroke (NINDS) Parkinson’s Disease Biomarkers Program (PDPB), the Michael J Fox Foundation Parkinson’s Progression Markers Initiative (PPMI) and BioFIND study, and the Harvard Biomarkers Study.
Samples include cerebrospinal fluid (CSP), RNA, blood plasma and DNA samples from more than 3,000 patients and more than 1,700 healthy controls.
“These initiatives have highlighted a shared interest between public and private organisations, and the data being generated present an opportunity to drive biomarker development more intensively than to date for translation into more effective therapies,” the editorial said.
The biosample information could help scientists identify new biomarkers, but its huge scope has posed a challenge to both researchers and companies.
Despite major investments, companies have yet to come up with disease-modifying Parkinson’s therapies — those can delay progression or relapses. Collaboration may be the way to achieve breakthroughs.
The Accelerating Medicines Partnership researchers will be digging into the large amounts of data to identify the most promising biomarkers of disease progression, which can then be used to validate new treatments.
Importantly, both the data and analyses will be shared with the broader research community in a platform called the AMP PD Knowledge Portal.
The portal will also incorporate data from two ongoing Phase 3 clinical trials in patients with early Parkinson’s disease. One is the Study of Urate Elevation in Parkinson’s disease (SURE-PD3; NCT02642393) and the other the Efficacy of Isradipine in Early Parkinson Disease (STEADY-PD3; NCT02168842).
In addition, the portal will enable the rest of the research community to participate in the collaborative effort and share their results.
“Sharing results from analyses of Phase 3 data via the knowledge portal will provide the opportunity to conduct analyses on a scale that could not be performed by a single partner alone,” according to the editorial.
The program will have three stages. The first, expected to end within 18 months, will create the working groups, the knowledge portal, and further analyze RNA from patients to identify potential biomarkers.
The second stage, expected to take three years, will pursue additional large-scale biomarker discovery to categorize patients, monitor their disease progression, and predict their outcomes. It will also identify Parkinson’s biomarkers that are common among cerebrospinal fluid, plasma, and brain tissue.
The last stage will validate the most promising biomarkers.
NIH will manage the partnership with GlaxoSmithKline, the Michael J Fox Foundation, NINDS, Pfizer, Sanofi, Celgene, Verily Life Sciences and the FDA, and coordinate the pledged investment of $24 million over five years.
“Bringing together the collective capabilities and resources across public and private sectors offers the best opportunity to identify and address challenges in early-stage drug development and thus the best opportunity for finding a cure,” the editorial concluded.