Validation of USP30 as a Therapeutic Target for Parkinson's Disease Modification
Target Advancement Program, 2017
Mitochondria provide energy for neurons to function and survive. Evidence suggests that individuals with Parkinson's disease (PD) have poorly functioning mitochondria, such that the energy demand is not met and toxic products build up and cause damage to neurons. USP30 is a newly discovered protein that regulates the clearance of poorly functioning mitochondria by modifying levels of a protein called ubiquitin.
This study will attempt to identify candidate biomarkers (track disease activity) of USP30 through testing USP30 inhibitors in proof-of-concept systems to enable effective biomarker use during USP30 inhibitor clinical development.
We will test USP30 inhibition on poorly functioning mitochondria to see if mitochondrial function and neuron health is improved. We will also develop profiles of USP30 inhibition activity, such as profiles of ubiquitin levels, gene expression and dopamine metabolism. In addition, we will validate antibodies (immune system proteins) against USP30 that may then be used as a standard for a variety of research applications.
Impact on Diagnosis/Treatment of Parkinson's disease:
Demonstration of beneficial effects on PD neurons after USP30 inhibition is an important step toward justification of USP30 as a valid target for Parkinson's disease drug development.
Next Steps for Development:
Mission Therapeutics is conducting studies in pre-clinical models of Parkinson's disease. If positive data comes from theses studies, Mission may consider progression toward fully-enabled clinical development of USP30 inhibitors for Parkinson's disease.
Medical Director, CNS Translational Medicine at Mission Therapeutics
Location: Cambridge, United Kingdom
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