- Five research teams have been granted $9.75 million in total funding through The Michael J. Fox Foundation’s ongoing efforts to validate quantitative biomarkers for Parkinson’s disease
- All funded teams aim to produce “quantitative” biomarkers, which measure and track the progression of PD through biology, to be ready for use in drug trials within two to three years
- Quantitative biomarkers would improve drug trials, especially those testing therapies to slow or stop the progression of PD, by giving specific, objective measures tied to disease progression
NEW YORK (May 30, 2024) — The Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced today $9.75 million in total awards to five research teams working on tools to biologically measure and track the progression of Parkinson’s disease (PD) from its earliest detection through more advanced stages. Collectively referred to as “quantitative” biomarkers, these tools would improve clinical trials with clear, objective measures.
Parkinson’s trials have historically relied on subjective assessments to judge the speed at which a person’s PD is progressing. Those assessments come from clinician observations or trial participants filling out surveys noting their symptoms, but they can be inconsistent based on the visit, clinician or person. These inconsistencies make it difficult to clearly judge whether a therapy is effectively helping the individual living with disease. To objectively test if a therapy can slow or stop the progression of Parkinson’s and bring functional benefit to patients, we need validated and reliable tools to measure the biology associated with that progression.
Current biomarker-based approaches do not provide the level of detail needed to assess the effect of a given therapy. The biomarker test that was validated last year, the alpha-synuclein seeding amplification assay (aSyn SAA), is binary, meaning it shows whether or not the signature misfolded protein in PD (alpha-synuclein, or aSyn) is present. It does not reveal how much of the protein is present or whether the amount is increasing or decreasing over time.
In contrast, quantitative biomarkers would be capable of providing specific measurements related to disease progression, equipping clinical trial researchers with powerful indicators of whether an experimental treatment is having an effect.
“Our Foundation exists to accelerate the development of better treatments for people with Parkinson’s, and better treatments come from better clinical trials. Quantitative biomarkers would empower these trials in a significant way, giving us our best view yet of a treatment’s efficacy.,” said Todd Sherer, PhD, MJFF’s chief mission officer. “The projects announced today aim to fast-track clinical trial science to move us meaningfully closer to our goal of curing Parkinson’s disease.”
Funded Research Projects for Quantitative Biomarkers
The quantitative biomarker funding goes to researchers developing some of the most promising tools to biologically detect, measure and track the progression of PD. Five research teams received support for their work. MJFF continues to review applications and plans to award funding to additional teams by the end of 2024.
Notably, all of the funded projects hold potential to deliver clinical trial-ready quantitative biomarker tests within the next two to three years, just when many of the therapies in the Parkinson’s pipeline currently could reach Phase III testing, a crucial juncture for drug makers needing to determine a therapy’s effectiveness.
Biochemical biomarker research projects
Three of the funded research teams are focused on developing reliable, lab-based tests to measure the amount of misfolded aSyn in readily available biosamples, including skin and blood. They include:
- Kalpana Merchant, PhD, will lead a consortium of groups focused on blood-based SAA, collaborating with Daniela Berg, MD, at University Hospital Schleswig-Holstein Kiel, Nobutaka Hattori, MD, PhD, at Juntendo University, Johan Skog, PhD, at Exosome Diagnostics, Erez Eitan, PhD, at NeuroDex, Anton Iliuk, PhD, with Tymora Analytics and Luis Concha, PhD, at Amprion in leading an effort to validate a blood-based quantitative biomarker for SAA.
- A team co-led by Concha and Tatiana Foroud, PhD, at Indiana University is working to validate a skin-based quantitative biomarker from SAA.
- Tuomas Knowles, PhD, and his lab at Cambridge University are developing microfluidics-based technology to improve the speed of SAA testing, which is currently conducted in spinal fluid, and make it quantitative.
Imaging research projects
Two funded research teams are developing imaging tools to detect clumps of aSyn in the brain, using positron emission tomography (PET) imaging technology. They include:
- David Russell, MD, PhD, from Invicro with the support of Makoto Higuchi, MD, PhD, are heading up a collaboration between the National Institute of Radiological Sciences in Japan and Invicro to develop a tool to visualize aSyn in the brain and track how it changes over time.
- Led by Helen Mitchell, PhD, and Eric Hostetler, PhD, Merck is developing its own tool to visualize aSyn in the brain and track how it changes over time in PD.
The Merck team was last year’s winner of MJFF’s Ken Griffin Alpha-synuclein Imaging Competition. The $10-million competition — funded by a leadership gift from Ken Griffin, founder and CEO of Citadel and founder of Griffin Catalyst — launched in 2019 in pursuit of an imaging tracer to visualize misfolded aSyn in the living brains of people with PD. This scientific race helped catapult biomarker research forward.
Details on the funded studies, including grant abstracts and researcher bios, are available on the Foundation’s website, www.michaeljfox.org.
About The Michael J. Fox Foundation for Parkinson’s Research (MJFF)
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding $2 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; creates a robust open-access data set and biosample library to speed scientific breakthroughs and treatment with its landmark clinical study, PPMI; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us at www.michaeljfox.org, Facebook or Twitter.
Media Contact:
Kristina Magana
The Michael J. Fox Foundation
kmagana@michaeljfox.org
212-509-0995