Investigating Mitophagy as a Therapeutic Intervention for Parkinson’s Disease

Presenter: Ciara Selders

Research Category: Physical and LIfe Sciences
Award: Scholarship

Parkinson’s Disease is a multisystem neurodegenerative condition that currently has no disease-modifying therapy available. Tens of thousands of Americans are diagnosed with Parkinson’s each year, and the estimated cost of disease treatment and financial impact on patients stretches into the tens of billions each year in the United States. As a member of Amgen’s Neuroscience department, my goal is to support the development a small-molecule drug that can promote the health of dopamine neurons through the encouragement of a mitochondrial recycling process known as mitophagy. By optimizing and performing assays using novel Amgen drugs, I am able to provide the program with various readouts of how these compounds work in neurons under different conditions. In order to simulate the mitophagy process, neurons are treated with various insults that will damage mitochondria within each cell. Two insults in this experiment, Oligomycin A and CCCP, reduced mitochondria levels by approximately 50% after 24 to 48 hours after treatment. This result demonstrates that mitophagy is successfully occurring, allowing for a suitable in vitro model to test Amgen compounds. Creating a therapy to target mitophagy will hopefully provide patients with the first-ever disease-modifying therapeutic for Parkinson’s. Enabling mitophagy to occur will allow the revival of neurons by replenishing the cell with functional mitochondria. This has great potential to fulfill a largely unmet therapeutic need in the Parkinson’s community. By preventing or delaying serious Parkinsonian symptoms, the patient has a chance to live without serious medical assistance for a prolonged period of time.