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Using Neural Cells to Test New Drugs that Activate PINK1 and Parkin

Promising Outcomes of Original Grant:
Even though most people with Parkinson's disease (PD) do not have a genetic mutation -- a change in their DNA -- that causes the disease, genes can influence the risk of developing PD and its course. Earlier, we sought to identify genes that influence PD progression. By comparing genes of individuals with more and less severe Parkinson's, we identified the genes that may influence the symptoms. We are currently aiming to confirm this finding in neural cells originating from the stem cells of individuals with PD. Additionally, we are developing powerful new computer techniques to analyze genetic data from people with PD. We expect these techniques to improve our ability to identify genetic causes of disease.

Objectives for Supplemental Investigation:
Two proteins, PINK1 and Parkin, prevent cell death by breaking down damaged mitochondria, cell's energy generators. Mutations in PINK1/Parkin genes that render these proteins unable to perform their function cause PD. In this project, we will generate neural cells from the stem cells of people with Parkinson's caused by PINK1/ Parkin mutations. As a result, the neural cells will have these mutations as well. We will then evaluate the condition of the neural cells and their ability to break down damaged mitochondria. These cells with serve as a model for the testing of PINK1/ Parkin-activating drugs. While in the original project we aim to identify and confirm new genes that influence Parkinson's symptoms, in this new project, we will develop a way to test drugs that activate these two genes involved in PD.

Importance of This Research for the Development of a New PD Therapy:
In Parkinson's disease, damaged mitochondria are not removed from the cell. Restoring the removal processes may help alleviate the symptoms and slow the disease progression. In this project, we will develop a platform for testing of PINK1/ Parkin-activating drugs, which may potentially restore the removal process and treat Parkinson's disease. We and other researchers believe that therapies that target these genes could help many people with PD, even though the vast majority of people with Parkinson's do not have mutations in the Parkin and PINK1 genes.


  • Steven Finkbeiner, MD, PhD

    San Francisco, CA United States

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