The U.S. National Institutes of Health is starting a pioneering, multi-pronged public-private partnership program to try to accelerate the development of Parkinson’s treatments.
What makes it special is how big it is. The NIH-led collaboration includes government, pharmaceutical companies, life science companies and non-profit organizations. The partners are calling the collaboration AMP PD — for Accelerating Medicines Partnership in Parkinson’s Disease.
The initial focus will be identifying biomarkers of Parkinson’s progression and assessing their potential as targets for therapies.
“Advancing treatments for Parkinson’s disease is hampered by insufficient understanding of biological networks,” NIH Director Francis S. Collins said in a press release. “Drugs aimed at seemingly promising therapeutic targets fail in clinical trials.”
“By combining our expertise and resources,” the partners “hope to increase our collective odds of success in accelerating the development of effective treatments for a million Americans who suffer from this debilitating disease,” he added.
Although scientists have been doing a lot of Parkinson’s research and conducting a number of clinical trials of potential therapies, regulators have yet to approve a drug that can modify the course of the disease.
Partners in the new collaboration include some of the best-known players in Parkinson’s work: Celgene, GlaxoSmithKline, The Michael J. Fox Foundation for Parkinson’s Research, Pfizer, Sanofi, and Verily.
The partners will chip in on the program’s financing to generate $12 million over five years. The Foundation for the National Institutes of Health will manage the program.
“The expansion of AMP [the Accelerating Medicines Partnership] into Parkinson’s disease is a testament to the success of this groundbreaking initiative that is radically changing the way we approach early-stage drug development,” said Maria C. Freire, president and executive director of the Foundation for the National Institutes of Health. The foundation “is proud to play a role in this partnership by harnessing the collective capabilities and resources from the public and private sectors with the aim of advancing biomedical research and ultimately improving health,” she said.
The NIH’s National Institute of Neurological Disorders and Stroke expects to match the private-sector investment — and add $12 million if funds are available. The U.S. food and Drug Administration will also be a key partner, providing regulatory guidance for drug development.
One of the program’s major benefits is that all data and findings will be shared not only among the partners but also with the entire research community.
Verily is developing an AMP PD Knowledge Portal to facilitate the sharing. The portal will give researchers access to data from studies that covered more than 3,000 Parkinson’s patients and 1,700 healthy controls.
They can use the data to try to identify which biomarkers work best at predicting Parkinson’s progression and patients’ outcomes. The Michael J. Fox Foundation and the National Institute of Neurological Disorders and Stroke funded the studies.
The portal should lead to an unprecedented amount of genetic analyses that the partners would be unable to conduct alone, the collaborators said.
“There is a wealth of biosamples and data already collected by NIH and MJFF [the Fox foundation] from people with Parkinson’s disease,” said Walter Koroshetz, director of the National Institute of Neurological Disorders and Stroke. “Sharing resources from public-private partnerships to generate and analyze ‘big data’ made available through AMP may be our greatest opportunity for accelerating the pace of discovery for translation [of research] into more effective treatments” for Parkinson’s, he said.
“The AMP PD Knowledge Portal will provide data storage, pipelines and visualization tools that could enable unique opportunities for data science solutions for human disease modeling and for the identification of the underlying biology related to PD [Parkinson’s disease] pathogenesis,” said Margaret Sutherland, the program director of the National Institute of Neurological Disorders and Stroke and co-chair of the AMP PD partnership steering committee.