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Developing Cell-Based Tools for the Evaluation of LRRK2 Inhibitors

Promising Outcomes of Original Grant:
Mutations in the LRRK2 gene are a leading genetic cause of Parkinson's disease (PD) and cause dysfunction in the LRRK2 protein. We have previously demonstrated that cells donated by people with LRRK2-associated PD differ from those donated by healthy people. In addition, such differences were also observed in cells from some people with PD with an unknown cause. These differences affected some key properties of the cells and were reverted by drugs called LRRK2 kinase inhibitors. Thus, such cells may be useful in clinical trials as experimental tools for studying the efficacy of LRRK2 kinase inhibitors.

Objectives for Supplemental Investigation:
The cells used in our previous studies were transformed into nerve cells from stem cells. We now aim to determine whether our findings can be observed also in non-transformed cells from people with LRRK2-associated and other forms of PD. Non-transformed cells are easier to obtain and work with than transformed cells. Changes in key cellular properties will be correlated with changes in the function of the LRRK2 protein. We also aim to determine the mechanism of these cellular changes.

Importance of This Research for the Development of a New PD Therapy:
Highly specific LRRK2 kinase inhibitors have been developed. The efficacy of kinase inhibitors will be easier to measure in clinical trials using easily obtainable non-transformed cells from people with Parkinson's disease.


  • Sabine Hilfiker, PhD

    Newark, NJ United States

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