Today there are more therapies to slow or stop Parkinson's in human trials than ever before. But those treatments face challenges in proving their effectiveness, and we want to ensure that even more potential therapies enter clinical testing.
Objective, biological markers of Parkinson's disease -- called biomarkers -- would be a game-changer. A perspective published today in the journal Science Translational Medicine lays out the needs and opportunities in Parkinson's biomarker research.
Nearly 40 experts, including Michael J. Fox Foundation (MJFF) CEO Todd Sherer, PhD, and several other senior staff, write that "the creation of tools enabling development of disease-modifying therapies in Parkinson's is a reachable, immediate goal, worthy of research investment." Indeed, MJFF invests significantly in the development of Parkinson's biomarkers, and the ideas in the paper originated from a Biomarkers Discovery Workshop convened by our Foundation in March 2016. The work was also supported and further developed at a meeting held by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health.
“Parkinson’s disease patients urgently need better treatments, and NIH-funded scientists are continually unraveling the biologic basis of the disease. However it takes a truly collaborative effort to deliver on the promise of the science,” said Walter J. Koroshetz, MD, NINDS director. “Our hope is that by working together through public-private partnerships we can accelerate the pace researchers can find effective Parkinson’s disease biomarkers and better treatments for patients.”
Speeding New Treatments to Patient Hands
The many potential therapies in human testing -- against Parkinson's protein alpha-synuclein, for example, and repurposed from other diseases -- create an urgent need for Parkinson's biomarkers. Most of these trials are following people in the years after diagnosis to see if a drug slows progression of the disease.
The challenge is that people with Parkinson's progress at different rates, so showing a treatment effect is difficult. You may need hundreds of participants observed over multiple years, and a false negative is still possible. If scientists had biomarkers that predicted who would progress faster, they could see an effect sooner and require fewer participants and a shorter study.
The perspective authors point out that the MJFF-led Parkinson's Progression Markers Initiative (PPMI) study shows distinct trajectories of movement symptom decline in early disease, with some progressing much faster than others. Our Foundation is funding PPMI researchers and others to look for biological predictors of those trajectories to help trials choose the fast progressors to test new treatments.
Building on What We Have Now
Some trials are using brain scans of dopamine activity (DAT scans) to help select participants, and last month the European Union's regulatory agency endorsed DAT scan as a tool to enrich Parkinson's trials. However, it's not clear if DAT predicts a person's speed of progression. It shows if someone is experiencing the Parkinson's effect of dopamine loss (as opposed to only exhibiting the clinical symptoms).
It's likely, the perspective authors write, that there will not be one Parkinson's biomarker but a mix of multiple markers that help scientists understand and predict one's experience with disease.
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