
Pioneering research is underway to develop a drug that could slow the development of Parkinson’s disease.
The project is a partnership between Parkinson’s UK and the University of Sheffield, and is backed by up to £1.2million in funding from the charity.
The research aims to create a world-first drug that can protect the dopamine-producing brain cells that are lost in Parkinson’s by targeting mitochondria.
Research has shown that in Parkinson’s the brain cells affected have faulty mitochondria. This means they can’t produce enough energy and is believed to be one of the main drivers of the condition.
Dr Heather Mortiboys, a Parkinson’s UK senior research fellow at the University of Sheffield, has identified molecules with excellent mitochondrial restoration properties.
Now, Dr Mortiboys is collaborating with Parkinson’s Virtual Biotech to take these molecules forward.
The ultimate aim is to create a drug that can boost the function of mitochondria to halt Parkinson’s, something no treatment can currently do.
Over the next 12 months, the team will develop and test the drug-like molecule in cells from people with Parkinson’s.
If this is successful, the molecules will then move forward into further testing in animal models before moving into clinical trials in people with Parkinson’s.
“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,” says Dr Mortiboys, a senior lecturer in the University of Sheffield’s SITraN.
“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, says: “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 groundbreaking academic researchers in the UK and taking their discoveries from the lab towards clinical trials.
“With 145,000 people living with Parkinson’s in the UK, 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.”








