Edinburgh researchers join new £10m centre to transform search for Parkinson’s treatments

Researchers and people with Parkinson’s in Scotland are joining forces at a new £10 million research centre, in a push to turn decades of discovery into treatments that could transform lives.

The UK Dementia Research Institute Parkinson’s Research Centre – jointly funded with Parkinson’s UK – launches today and connects research teams at the University of Edinburgh, the University of Oxford and University College London to tackle one of medicine’s most stubborn challenges – why, despite scientific advances, there is still no treatment that can slow or stop the condition progressing.

Together, the teams will investigate why Parkinson’s develops, why it progresses and how cutting-edge science can deliver better diagnosis and treatment.

Parkinson’s affects around 166,000 people in the UK, including 14,000 people in Scotland. Someone is diagnosed every 20 minutes.

The centre is led by Professor Miratul Muqit, a practising neurologist and internationally recognised Parkinson’s researcher based at the University of Edinburgh, whose work has helped reveal how changes in key genes affect the health of brain cells.

Discoveries from this field are helping pave the way for targeted therapies now being tested in early-stage clinical trials.

Professor Miratul Muqit, Director of the UK Dementia Research Institute Parkinson’s Research Centre, said: “We know more about Parkinson’s than ever before – but people living with the condition are still waiting for effective treatments that can slow, stop or prevent it.

“This centre is built to change the pace of progress. By connecting leading teams across Edinburgh, Oxford and London, we can bring different parts of the Parkinson’s puzzle together – from genes and brain cells to brain circuits and symptoms.

“Our ambition is to make this centre a beacon for open, collaborative science.”

Professor David Dexter, Director of Research at Parkinson’s UK, said: “For people living with Parkinson’s, better treatments cannot come soon enough. That is why this centre, and the collaborative philosophy at its heart, is so important.

“It puts people with Parkinson’s alongside world-class researchers, helping make sure the science is focused on the questions and symptoms that could make the biggest difference to everyday life.”

People with Parkinson’s have helped shape the centre’s direction from the very beginning – including sitting on the interview panels that appointed its first research leaders.

Shafaq Hussain-Ali, who has Parkinson’s, was part of the panel that selected the centre’s Group Leaders. He said: “It was a privilege to be involved. The new centre recognises that Parkinson’s research cannot happen without the involvement and participation of the Parkinson’s community.

“Hearing from the researchers about their passion for transformative science has filled me with hope about what is to come.”

Project aims to speed up delivery of treatments for motor neuron disease

A new project by researchers in Edinburgh aims to identify combinations of existing drugs that could be used together to treat motor neuron disease (MND).

Led by Prof Siddharthan Chandran, Group Leader at the UK Dementia Research Institute (UK DRI) and Director of the Euan MacDonald Centre for MND Research, both at the University of Edinburgh, the £3.3 million project is funded by the medical research charity LifeArc, as part of an ongoing partnership between the charity and the UK DRI.

The partnership brings together the strengths of UK DRI’s research into discovery science with LifeArc’s translational expertise to take exciting lab discoveries forward and translate them into tangible benefits for patients.

MND is a life-limiting condition that causes progressive weakness of the muscles due to the degeneration of motor neurons in the brain and spinal cord. There is currently only one drug approved to treat the disease in the UK, riluzole, which has only a modest effect.

There is an urgent unmet need for effective therapies to treat MND. But the brain is complex, and targeting one biological pathway with a single drug might not be enough to slow down or stop the degeneration of motor neurons.

The new project seeks to drastically accelerate the development of new treatments by identifying existing drugs which target multiple disease mechanisms implicated in MND.

Typically, new drugs can take up to 15 years to progress through development and clinical trial stages, but with this approach, treatments could be tested in the clinic within four years.

Prof Siddharthan Chandran, Group Leader at the UK Dementia Research Institute (UK DRI) and Director of the Euan MacDonald Centre for MND Research, both at the University of Edinburgh, said: “As has been shown for cancer therapy, using combinations of drugs that target different pathways might be our best chance of slowing or stopping the progression of MND.

“This innovative project is an important next step in identifying effective medicines for MND.”

In the first stage of the study, the researchers will prioritise the top drug candidates, using both laboratory-based tests on motor neurons grown in the lab from patient donated stem cells, and a machine-learning, artificial intelligence approach to review published scientific studies of MND.

Next, the top candidate drugs will be tested in pairs in combination in the stem cell models of MND, against different biological pathways known to be implicated in MND.

The ultimate goal is to seek regulatory approval to test the most promising and effective combinations of drugs in the Euan MacDonald Centre’s MND-SMART (Motor Neuron Disease – Systematic Multi-arm Adaptive Randomised Trial) trial.

This pioneering trial across 20 sites in the UK is designed to shorten the time it takes to find medicines that can slow or stop MND. Unlike typical clinical trials which test a single treatment, MND-SMART is testing several treatments at the same time. It is also an adaptive trial which means that new drugs can be added, and those proven ineffective can be dropped.

This new project complements another recently announced MND initiative, EXPERT-ALS, which aims to rapidly identify promising drug candidates in small scale trials, before definitive evaluation in Phase 3 platform trials such as MND-SMART.

Dr Paul Wright, MND Translational Challenge Lead at LifeArc said: “Our involvement in this research is part of an ambitious long-term £100m funding programme we have launched to help tackle neurodegenerative conditions and find treatments where none currently exist. 

“By working with UK DRI we are uncovering promising life science research, like Professor Siddharthan’s, that we can support with funding or by offering our scientific resources and expertise in translational research.

“Ultimately, our aim is to accelerate the process of finding medical breakthroughs that can prevent and stop these life-threatening diseases progressing.”