MJFF funds biomarker research to improve Parkinson’s clinical trials

Nearly $10 million will be distributed among five research teams

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded $9.75 million to researchers working on validating new biomarkers for tracking Parkinson’s disease progression.

The funds are split across five different research teams, each of which is looking for quantitative biomarkers that will be ready for use in clinical trials within the next two to three years. The foundation believes such biomarkers could help improve clinical research by offering a clear and objective measure of disease progression beyond the subjective clinician- or patient-rated measures currently used.

“Our Foundation exists to accelerate the development of better treatments for people with Parkinson’s, and better treatments come from better clinical trials,” Todd Sherer, PhD, MJFF’s chief mission officer, said in a foundation press release. “The projects announced today aim to fast-track clinical trial science to move us meaningfully closer to our goal of curing Parkinson’s disease.”

In Parkinson’s, the progressive loss of nerve cells that produce the important signaling chemical dopamine lead to movement problems and an array of other nonmotor symptoms. Underlying this neurodegeneration is the toxic accumulation of a misfolded version of the alpha-synuclein protein. Levels of the abnormal protein increase over time as the disease progresses.

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Need for objective biomarkers in Parkinson’s disease

Currently, Parkinson’s clinical trials rely on subjective assessments of symptoms to see how fast the disease is progressing and whether an experimental treatment is slowing it down. These can include patient-reported surveys or clinician-rated assessments.

However, these outcome measures can be inconsistent with each visit or depend on the person making the ratings, creating a need for objective measures that are based on the underlying biology of Parkinson’s and accurately reflect the disease’s course.

There are already some biomarker tests that are believed to be useful for diagnosing Parkinson’s. Last year, MJFF led the validation of the aSyn SAA assay, a very sensitive test for detecting whether misfolded alpha-synuclein is present in a spinal fluid sample.

While simply saying whether the protein is present can aid a Parkinson’s diagnosis, such a test lacks the level of detail needed to track how the disease is progressing over time or with a certain treatment in clinical testing.

To do so, a test would have to be quantitative, measuring how much alpha-synuclein is present and evaluating how that’s changing with the disease’s progression.

The funded projects are all aimed at identifying new quantitative biomarkers for Parkinson’s clinical research that will overcome these limitations.

“Quantitative biomarkers would empower these trials in a significant way, giving us our best view yet of a treatment’s efficacy,” Sherer said. 

Three projects are focused on lab-based tests to measure the amount of misfolded alpha-synuclein in readily available biological samples such as the skin and blood. The other two are developing positron emission tomography imaging tools that could detect alpha-synuclein clumps in the brain and track how they change over time. One project is being conducted at Merck, which won MJFF’s Ken Griffin Alpha-Synuclein Imaging Competition last year.

More details about the recently funded studies and past awardees can be found on the foundation’s website. Meanwhile, MJFF is continuing to review applications and plans to award funding to more research teams by the end of the year.