4 Fox Focus | Research
The Next Step into the New Era :
A Framework for Applying Parkinson ’ s Biology to Trials
By Jen Fisher Wilson
For decades , researchers were limited to visible , clinical symptoms to detect Parkinson ’ s and assess whether treatments worked . But the discovery of the alpha-synuclein biomarker has changed everything because with it , they can see into PD ’ s underlying biology . Now a new research framework builds on the biomarker ’ s potential by defining and staging PD based on its biological underpinnings for the first time .
The framework , previewed in our last issue of The Fox Focus on Parkinson ’ s and published in January in The Lancet Neurology , takes PD another major step into its new biological era . This tool for researchers is expected to evolve with advances in understanding and tracking the disease , yet , already , drug developers see it accelerating the timeline to better treatments .
That ’ s because with a research framework for Parkinson ’ s , biopharma companies can more efficiently test novel therapies in individuals who have the targeted disease biology , and regulators can better assess the impact of these therapies on disease progression . A similar biological framework in Alzheimer ’ s disease led to successful trials and new drug approvals , with the first drugs to slow Alzheimer ’ s-related cognitive decline appearing in 2022 and 2023 .
“ The success that the Alzheimer ’ s field has had with its biological framework provides the inspiration and motivation to achieve similar accelerated timelines in Parkinson ’ s ,” said Tanya Simuni , MD , lead author on the published paper and associate professor of neurology and director of the Parkinson ’ s Disease Movement Disorders Center at Northwestern University . “ Ten years from now , we hope we will look back and say this framework was the key that opened the door to next-generation treatments in Parkinson ’ s .”
Building on the Biomarker Breakthrough
The framework first defines Parkinson ’ s based on the presence of the pathological alphasynuclein protein that misfolds , clumps and damages the brain over the course of the disease . The ability to detect this pathology only became possible with the first major step into the biological era : the alpha-synuclein seeding amplification assay ( aSyn-SAA ) biomarker . Validated in April 2023 by The Michael J . Fox Foundation ’ s Parkinson ’ s Progression Markers