Study Rationale:
The diagnosis and treatment of Parkinson's disease (PD) would be aided by the availability of direct and highly sensitive tests for the abnormal clumps of protein alpha-synuclein, which have been linked to the disease. Two such tests have been developed by other laboratories, but these tests take from five to 13 days to perform in the laboratory. We have developed a similar test that takes less than two days to perform and allows us to measure the amount of alpha-synuclein clumps. In this study, we will further evaluate our new test using cerebrospinal fluid samples from people with PD and from healthy people. The biosamples will be provided by BioFIND, a clinical study sponsored by The Michael J. Fox Foundation for Parkinson's Research (MJFF) with support from the National Institute of Neurological Disorders and Stroke (NINDS).
Hypothesis:
We hypothesize that our new Parkinson's test, called alpha-synuclein RT-QuIC, will be highly accurate and much faster than similar tests.
Study Design:
The BioFIND samples will be sent to us blinded (coded), so that we do not know which samples came from people with PD and which from healthy people. We will test the samples and send the results back to MJFF/NINDS for decoding. Analysis of the results will allow us to determine the diagnostic sensitivity (the proportion of people with Parkinson's diagnosed correctly) and specificity (the proportion of healthy people who tested negative for Parkinson's). Sensitivity and specificity are two parameters commonly used to judge the performance of a test.
Impact on Diagnosis/Treatment of Parkinson's Disease:
If our new alpha-synuclein RT-QuIC maintains the diagnostic performance in our earlier studies, it should facilitate early and accurate diagnosis of PD. Such diagnoses may allow appropriately targeted treatments to begin earlier. In addition, such testing should improve clinical trials of new treatments by facilitating the selection of participants and allowing levels of abnormal alpha-synuclein to be compared in treated and untreated individuals over the course of a trial.
Next Steps for Development:
If the test results of the BioFIND samples are promising, we will expand to test even larger and more diverse sample sets to further evaluate the usefulness of the alpha-synuclein RT-QuIC testing of cerebrospinal fluid. We are also training other laboratories to perform their own alpha-synuclein RT-QuIC testing to encourage broader implementation and innovation.