Michael J. Fox Foundation Awards Mission Therapeutic, University of Oxford Grant To Test USP30 Inhibitor as Parkinson’s Treatment

Stacy Grieve, PhD avatar

by Stacy Grieve, PhD |

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The Michael J. Fox Foundation has awarded Great Britain’s Mission Therapeutics and the University of Oxford a grant to test the novel USP30 inhibitor on a range of disease models including Parkinson’s disease.

The New York-based foundation announced its grant, whose amount was not disclosed, in a press release on World Parkinson’s Day.

The grant will combine the potent USP30-targeted inhibitors of Mission Therapeutics and the stem cell-derived Parkinson’s models developed by Professor Richard Wade-Martins and his research group at the University of Oxford.

USP30 is a mitochondrial-associated deubiquitylating enzyme (DUB). The ubiquitin proteasome system is responsible for the degradation of proteins within cells.

More specifically, USP30 has been implicated in control of a process known as mitophagy, in which defective mitochondria are degraded and cleared from the cell. Under normal conditions, USP30 controls mitophagy. However, in neuronal cells affected by Parkinson’s, altered USP30 signaling can lead to the toxic accumulation of mitochondria, which damages neuronal cell. The hallmark feature of Parkinson’s is, in fact, the degenerate loss of neurons.

“USP30 is one of the more promising DUBs associated with mitophagy, in terms of published data and feasibility of compound development,” said Shalini Padmanabhan, PhD, the foundation’s associate director of research programs. “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.”

The Michael J. Fox Foundation is the world’s largest supporter of Parkinson’s research, having funded more than $700 million in research projects to date. D research.  To date, it has funded more than $700 million in research. It focuses on accelerating a cure for Parkinson’s as well as improving therapies for those already living with the disease.

Mission Therapeutics is an early-stage drug development company that selectively targets DUBs to treat neurodegenerative diseases, cancer and other illnesses with high unmet medical need. DUB inhibitors are gaining significant attention as an emerging drug class important for regaining control over protein homeostasis. The company is particularly interested in developing its class of USP30 inhibitors to better understand if and how these drugs may improve cellular resilience in Parkinson’s.

“Receiving funding from the Michael J. Fox Foundation is a great accolade,” said Michael Koslowski, MD, executive vice-president of research and development at Mission Therapeutics. “The collaborative study will provide key data that will guide the clinical development strategy of our USP30 inhibitor program. We are working hard to find new ways in which to tackle this difficult disease.”