Sheffield scientists have been awarded £1.2 million from the charity, Virtual Biotech Initiative, to develop a drug for the brain disorder Parkinson’s disease.
The drug will be developed to protect dopamine-producing brain cells to slow down the progression of the disease.
Researchers from the University of Sheffield’s Institute of Translational Neuroscience will be working together with the charity’s Virtual Biotech initiative to refine a molecule that can boost the function of the brain’s energy-producing mitochondria. This should halt Parkinson’s progression.
Parkinson’s disease is a brain disorder that leads to shaking, stiffness and difficulty with walking, balance and coordination. As a degenerative illness, symptoms gradually get worse over time.
The research will be led by Dr. Heather Mortiboys, who has been working with experts from Parkinson’s UK, to create the new drug.
She said: “Two more people are diagnosed with Parkinson’s disease every hour – that’s 18,000 people every year. Causing a loss of physical movement and uncontrollable tremors, the disease will affect 145,000 people in the UK alone in 2020.
“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. Mortiboys has previously identified two molecules with excellent mitochondrial restoration properties. This research was funded by a Parkinson’s UK Senior Fellowship grant.
Modifications were then made to these molecules, which maximise their ability to boost the mitochondria without causing any side effects.
These molecules will be tested in brain cells throughout the next phase of the project as well as investigating how the molecules work and what proteins they interact with inside the brain.
After a drug-like molecule is developed, tests to assess the effectiveness of this approach in animal models will occur before moving into clinical trials in people with Parkinson’s.
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.”
