6 Fox Focus | Research
Researchers Discover New Parkinson ’ s Biomarker ( continued )
a fluorescing agent that lights up if alphasynuclein clumps form . Normal alpha-synuclein is then seeded into the spinal fluid sample . If abnormal alpha-synuclein is present in the sample , clumps form and the dye lights up . If no abnormal alpha-synuclein is present , the dye doesn ’ t fluoresce .
After being tested in small , independent studies , in 2022 the assay was validated in the large , well-characterized cohort of the Parkinson ’ s Progression Markers Initiative ( PPMI ) study , MJFF ’ s landmark clinical study . The validation was carried out in 1,123 samples of spinal fluid contributed by PPMI participants over the years . The assay proved amazingly accurate , with 93 percent of participants with Parkinson ’ s having an abnormal test . ( Very few tests for neurologic disorders are over 90 percent sensitive for disease .) And , importantly , the test was abnormal in less than 5 percent of people without Parkinson ’ s .
The biomarker breakthrough , published in April in the scientific journal Lancet Neurology , was achieved by an international coalition of scientists led by The Michael J . Fox Foundation and PPMI .
Steady and critical advances in the pursuit of a reliable and accurate biomarker test have been the hallmark of PPMI , which was built for this purpose . The discovery enabled by the new test is the latest , and most significant , finding to date from the study .
Today , with this discovery in hand , Parkinson ’ s is moving from a disease primarily understood , diagnosed and measured through subjective clinical assessments to an objectively biologically defined disease — which makes possible new paradigms for clinical care , including earlier diagnosis and targeted treatments , and faster , smarter and cheaper drug development .
By helping to identify people at the earliest stages of PD , “ We could then study what happens at different biological stages of the disease ,” says Dr . Sherer . Says Ken Marek , MD , PPMI principal investigator , “ aSyn-SAA enables us to move to another level in effecting new strategies for prevention of disease .”
In addition to the new momentum in research sparked by the assay , progress on developing an imaging tool for alpha-synuclein , which marked significant progress last year , is simultaneously accelerating , with the MJFF-supported Ken Griffin Alpha-synuclein Imaging Competition spurring activity in imaging the protein in the living human brain .
MJFF ’ s urgent mandate is to drive the next stages of development of aSyn-SAA toward widespread and standard use as rapidly as possible . Since today the tool can elicit a binary response — showing that abnormal synuclein is either present or not — there is tremendous promise in quantification , in order to measure the amount of alpha-synuclein present . Additionally , an optimized assay would detect abnormal synuclein through a blood draw or nasal swab — a simple test that could be done in any doctor ’ s office .
“ I ’ m moved , humbled and blown away by this breakthrough , which is already transforming research and care , with enormous opportunity to grow from here ,” says Michael J . Fox . “ I ’ m so grateful for the support of patients , families and researchers who are in it with us as we continue to kick down doors on the path to eradicating Parkinson ’ s once and for all .”