Award Supports Project to Design Parkinson’s Drug

Researchers at the University of Sheffield have been awarded £1.2 million from Parkinson’s UK to continue with development of a drug that protects dopamine-producing brain cells in efforts to halt the degenerative disease.

Dopamine is a chemical which allows messages to be sent to the parts of the brain that help to coordinate movement. To do this effectively, the dopamine-producing brain cells need to be constantly active and they rely on mitochondria to function properly.

Scientists from the University’s Institute of Translational Neuroscience (SITraN) led by Dr Heather Mortiboys, have been working with experts from Parkinson’s UK to develop molecules that can boost the function of the brain’s energy-producing mitochondria.

Earlier work from Dr Mortiboys, funded by a Parkinson’s UK Senior Fellowship grant, identified two molecules with excellent mitochondrial restoration properties and the new award has enabled further progression of this work.

Dr Mortiboys said: “We’re delighted to continue our work with Parkinson's UK to refine these promising molecules to develop a treatment that could stop Parkinson’s in its tracks. The team has identified not only molecules which can restore mitochondrial function in dopaminergic neurons from people with Parkinson's, but also a novel mechanism by which they do this. 

“We’re progressing both a novel mitochondrial therapeutic target and novel molecules which act upon this target. The aim is to have a lead molecule which is active at the mitochondrial target, can restore mitochondrial function in dopaminergic neurons derived from people with Parkinson’s and is able to get into the brain in a whole organism.”

Dr Arthur Roach, Director of Research at Parkinson's UK, said: “We’re excited to fund the next stage of this full-scale project combining our own drug discovery know-how with experts from the University of Sheffield and two world-leading contract research organisations. Looking back, it’s incredible to see how this project had its origin in a small academic grant from Parkinson’s UK to screen for molecules that rescue defective mitochondria inside neurons. Since then, the charity and University have continued to work closely together to improve those initial compounds. 

“This new funding will greatly accelerate the project and shows our commitment to working with ground-breaking academic researchers in the UK and taking their discoveries from the lab towards clinical trials. There is a desperate need for new and better treatments and we hope this project will one day deliver a life-changing drug for people living with the condition.”

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