On World Parkinson’s day, Mission Therapeutics, a drug discovery and development company focused on selectively targeting deubiquitylating enzymes (DUBs) to treat neurodegenerative diseases, cancer and other diseases with high unmet medical need, announced that it has been awarded a grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF).
This research grant will support the testing of Mission Therapeutics’ potent and selective USP30-targeted inhibitors in translationally relevant stem cell-derived Parkinson’s disease models developed by Professor Richard Wade-Martins and his research group at the University of Oxford.
USP30, a mitochondrial associated DUB, has been implicated in the control of mitophagy – a process where dysfunctional mitochondria are selectively cleared from the cell. Failure of mitochondrial quality control may lead to degeneration of the highly active substantia nigra neurons in the brain, a pathological mechanism which results in Parkinson’s disease.
The inhibition of USP30 is being studied by Mission Therapeutics to see if this promotes mitophagy and thus improves cellular resilience in this and other neurodegenerative diseases. The objective of the research collaboration with Professor Richard Wade-Martins is to test Mission’s potent and selective USP30 inhibitors in a range of disease models - induced Pluripotent Stem Cells (iPSC)-derived from patients with sporadic and familial Parkinson’s disease.
Shalini Padmanabhan, PhD, Associate Director of Research Programs at MJFF:
“USP30 is one of the more promising DUBs associated with mitophagy, in terms of published data and feasibility of compound development. We hope that this collaboration between Mission Therapeutics and Oxford Parkinson’s Disease Centre will promote our understanding of the mechanisms and consequences of USP30 inhibition in Parkinson’s disease.”
Dr Michael Koslowski, Executive Vice President, Research and Development and Chief Medical Officer of Mission Therapeutics, added:
“Receiving funding from the Michael J. Fox Foundation is a great accolade, recognizing the quality of the research being done by Mission Therapeutics and Prof. Wade-Martins and his group. The collaborative study will provide key data that will guide the clinical development strategy of our USP30 inhibitor programme. We are working hard to find new ways in which to tackle this difficult disease, which is especially highlighted during this World Parkinson Awareness week, for patients and their families.”
Parkinson's disease is a chronic, degenerative neurological disorder that affects one in 100 people over age 60. There is no objective test, or biomarker, for Parkinson's disease, so the rate of misdiagnosis can be relatively high. Estimates of the number of people living with the disease therefore vary, but recent research indicates that at least one million people in the United States, and more than five million worldwide, have Parkinson's disease.