Promising Outcomes of Original Grant:
Certain forms of Parkinson's are due to defects in a cellular regulatory node known as the PINK1-Parkin pathway. With previous funding from MJFF, we sought to develop a probe technology to measure cellular activity involved in this pathway. Our initial approach proved to be unfeasible, so we explored an alternative application of our probe technology. It can provide a direct assessment of the functionality of this pathway and has potential as a biomarker for Parkinson's associated with the PINK1-Parkin. We were successful in measuring Parkin activity in model cell lines.
Objectives for Supplemental Investigation:
Our next steps are to establish the sensitivity of our measurement technology. We would also like to establish the reproducibility of the data we obtained previously. These initial experiments will be performed in model cell lines. We will next apply the assay to clinical samples and donor samples from people with and without Parkinson's.
Importance of This Research for the Development of a New PD Therapy:
Our studies could result in a biomarker to assess a person's susceptibility to Parkinson's disease. It could also be used in the development of treatments designed for Parkinson's patients with a defective PINK1-Parkin pathway. It could serve as an experimental platform to identify Parkinson's treatments that target Parkin activity. And it would allow clinical trial teams to identify which patients are most likely to benefit from such treatments.