Study Rationale: By the time the symptoms of Parkinson’s disease (PD) appear, many of the brain cells that produce the chemical dopamine have already died. This loss of dopamine-making cells leads to communication problems between the parts of the brain that help coordinate movement. The cells’ energy-producing mitochondria can also be damaged, further crippling the brain's communication network. Transferring healthy mitochondria to cells in which mitochondrial function is impaired has been shown to prevent cell death and may provide a novel approach to reversing the effects of neurodegeneration.
Hypothesis: Because mitochondrial damage contributes to PD pathology, we hypothesize that transplanting healthy mitochondria into affected brain cells might prevent neuronal degeneration and PD pathology.
Study Design: We will analyze the components of extracellular vesicles that are released from mitochondria (mitoEVs) in patients with PD. To do this, we will take samples of mitoEVs from healthy individuals and from people with PD of different ages and disease stages and study their contents. This analysis will allow us to generate a detailed picture of the different components of mitoEVs at various stages of PD. In addition, we will investigate whether healthy extracellular mitochondria can support mitochondrial function of neurons generated from people with PD.
Impact on Diagnosis/Treatment of Parkinson’s disease: If successful, translation of our findings into the clinic will allow the use of healthy mitoEVs to decrease symptoms and pathology in people with PD.
Next Steps for Development: Our findings will serve as a starting point for the development of new therapies that target mitochondrial dysfunction, including the exploration of mitochondrial replacement therapy as an option for treating PD.