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Soania Mathur, a former family physician, who has had Parkinson’s disease for over 25 years, believes she knows what’s best for her.
“As patients living with this disease, we really are experts in this disease, regardless of whether we have a medical background or scientific background or not,” she says.
The Foundation for the National Institutes of Health (FNIH) is amplifying patient voices like hers with a new addition to its innovative Accelerating Medicines Partnership (AMP) initiative: a Parkinson’s Disease and Related Disorders (AMP PDRD) program.
AMP PDRD is the second iteration of the FNIH’s original program, AMP Parkinson’s Disease (AMP PD).
“At the FNIH, we’ve been working on Parkinson’s disease for five years and we just completed the first phase of our Parkinson’s initiative,” explains Dr. Alessio Travaglia, Director of Translational Science, Neuroscience, at the FNIH. “This second iteration asked, What should we do now? What is the next big question that we need to address? And we decided that we needed to understand the differentiation between multiple similar diseases, thus creating the AMP PDRD program.”
“Lewy body dementia, progressive supranuclear palsy, multiple system atrophy, these are all similar disorders that follow a similar pathology—but there are some differences,” adds Steve Hoffmann, Vice President of Science Partnerships at the FNIH. “There are differences in biomarkers, proteins, and the symptoms that occur in each, so understanding those differences will be critical to not only develop a deeper understanding of what Parkinson’s disease is, but also of what Parkinson’s disease is not, as well as how we can effectively treat those diseases, either in a common way or in a separate individual way.”
A legacy of collaboration
The AMP PDRD program is the 12th program from FNIH’s Accelerated Medicines Partnership, a public-private partnership between government, academia, industry, advocacy, and patients to advance new targets, understanding of disease, and sharing of data and samples. The FNIH’s AMP programs facilitate collaboration between researchers, industry, government, and advocacy organizations to solve some of the most pressing problems in healthcare today.
“The FNIH’s role as a neutral convener has been an essential element to the success of programs like the Accelerated Medicines Partnership,” says Hoffmann. And the FNIH’s AMP PDRD follows a similar model to other FNIH programs in that it not only welcomes patients and patient advocates for different diseases into the program but also gives those patients and advocates a consequential voice in the design and development of the program.
“There remains such a broad heterogeneity of disease types,” notes Hoffmann. “As patients often say, My arthritis is not your arthritis. My Parkinson’s is not your Parkinson’s. That patient voice has become more and more important, not only in the early design of these programs, but as we go through the process and welcome in partners and experts at every level.”
“When I was first diagnosed, there wasn’t much of any kind of patient voice that was recognized,” Mathur says. “It was not uncommon at all for me to meet researchers that had never met someone with Parkinson’s disease, yet their life’s work was dedicated to this field. So it’s really been a great change that’s happened over the last few years, where patient voices are not just tokenistic, but they’re actually valued.”
And as Mathur explains, patient anecdotes and input will ultimately only improve research models.
“I think all areas of research can really benefit from patient involvement,” from research to clinical trial design to post-study communication, she says.
Goals of the AMP PDRD program
Like other AMP programs, the AMP PDRD program is focused on breaking the silos of research on Parkinson’s in order to reach larger goals.
“We have done this very well and successfully across multiple diseases of high need in the past: Alzheimer’s disease, heart failure, autoimmune and immune-mediated diseases, our earlier iteration of the Parkinson’s disease program, and now in ALS as well,” adds Hoffmann.
And interestingly, as the FNIH builds its multiple repositories of data, collaboration, and research deliverables across a broad molecularization of disease, they are simultaneously building an even more vast compendium of information that researchers can interrogate with powerful analytic tools, like AI, to cross-examine multiple diseases, possibly finding overlap and solutions to treatment that would previously have been unknown.
In the meantime, one of the immediate goals of AMP PDRD is developing the proper patient populations.
“There are two main focuses when it comes to how we are trying to tackle things like neurodegenerative diseases,” says Travaglia. “One is to have a homogeneous population for clinical trials. We are getting toward personalized medicine, and so rather than having a drug cure it all, we are working to understand what population we have in hand, make sure we find a homogeneous set of patients, and treat those patients rather than trying to cure it all at once, because if we try to cure it all at once the chances of success are going to be very slim. The other thing that is happening a lot in the field is trying to research earlier in the disease versus treating the patients in the later stage of the disease.”
