A Sheffield academic has been awarded £100,000 of funding by the Virtual Biotech Programme - the drug development arm of charity Parkinson's UK - to develop a new treatment that could protect brain cells affected by Parkinson's disease, potentially slowing, or stopping its progression for the first time.
Dr Heather Mortiboys and her team from the University of Sheffield's Institute for Translational Neuroscience (SITraN) and the University's new Neuroscience Institute will work with the charity Parkinson's UK to modify a number of drug compounds that have been found to boost cell function in people living with Parkinson's.
Dopamine-containing brain cells - vital for healthy coordination and movement - rely on energy-producing mitochondria to function, but in people living with Parkinson's the mitochondria, or powerhouse of the cells are disrupted and the cells begin to fail and slowly die.
As a Senior Research Fellow for Parkinson's UK, Dr Mortiboys and her team have identified a number of drug compounds which could boost the function of these dopamine-containing brain cells. Their previous research utilized recently developed methods to grow these brain cells from the skin cells of patients with Parkinson's disease, and importantly they developed a way to generate them in high numbers - something never achieved before - to test the identified drug compounds on these patient-derived cells.
Dr Mortiboys' research isolated a number of these compounds which were found to boost the mitochondrial function in these dopamine-producing brain cells and potentially reduce cell death; the cause of the main symptoms of Parkinson's which consist of loss of movement, tremors and rigidity.
Over the next 12 months, this award will enable Dr Mortiboys and her team to identify a lead molecule from the compounds which has the most beneficial effects on mitochondrial function of the brain cells, to be progressed along the drug discovery pipeline with partners at the National Institute of Health Research (NIHR) Sheffield Biomedical Research Centre.
It is hoped this new work will lead to the development of a treatment which will protect these brain cells, slow the progression of Parkinson's and extend the quality of life for people living with the disease.
Dr Heather Mortiboys, Institute for Translational Neuroscience,University of Sheffield:
The partnership between our team and Parkinson's UK could lead to a UK first in the development of treatments for Parkinson's, putting our research one step closer to pioneering a breakthrough treatment for Parkinson's patients."
"All the clinical treatments for people living with Parkinson's at the moment are based on easing these sometimes devastating symptoms," added Dr Mortiboys. "With this new funding award through the Virtual Biotech Programme, we have the potential to go on to develop a drug treatment which will actively address the root cause of these symptoms to slow, or halt the progression of Parkinson's for the first time."
The research forms part of the work of the University of Sheffield's Neuroscience Institute, that aims to translate scientific discoveries from the lab into pioneering treatments that will benefit patients living with neurodegenerative disorders.
Professor Dame Pamela Shaw, Director of SITraN and the Neuroscience Institute said: "There is an urgent need for treatments to protect the nerve cells that become damaged in patients with Parkinson's disease, which will have a crucial impact in slowing the progression of the condition and improving the quality of life.
"Dr Mortiboys and her team are identifying drugs which improve the function of, and reduce the damage to nerve cells in laboratory models of Parkinson's disease.
"We plan to take the most promising of these from the SITraN laboratories to progress along the drug discovery pipeline for patients with partners at the NIHR Biomedical Research Centre based at the Royal Hallamshire Hospital. We are hugely grateful to Parkinson's UK for supporting this important translational research."
Richard Morphy, Drug Discovery Manager at Parkinson's UK, said: "We are delighted to partner and work with Dr Heather Mortiboys and her team at the University of Sheffield. Through our Virtual Biotech initiative, we are committed to accelerating promising and breakthrough treatments for Parkinson's.
"This is an exciting new approach that could rescue defective mitochondria inside neurons to prevent dysfunction and degeneration of dopamine-producing brain cells. With 148,000 people living with Parkinson's in the UK, there is a desperate need for new and better treatments for Parkinson's. We hope the project will identify a superior group of molecules that could one day deliver a life-changing drug for people living with the condition."