Cookies on this website
We use cookies to ensure that we give you the best experience on our website. If you click 'Continue' we'll assume that you are happy to receive all cookies and you won't see this message again. Click 'Find out more' for information on how to change your cookie settings.

A collaborative study led by the OPDC's Dr Nora Bengoa-Vergniory has shown that compounds known as molecular tweezers could become a promising disease modifying therapy for Parkinson’s.

A team of researchers has shown that tiny compounds known as molecular “tweezers” could become a promising therapy to slow Parkinson’s. This new kind of drug works by pulling apart toxic clumps of protein that form in the brain during Parkinson’s.

The therapy has previously shown high potential for targeting toxic protein clumps that form in neurodegenerative conditions such as Alzheimer’s. The research teams therefore investigated whether a particular molecular tweezer, CLR01, was able to reduce formation of protein clumps in cell and mouse models of Parkinson’s.

The research was led by the Oxford Parkinson’s Disease Centre at the University of Oxford, created through funding from Parkinson’s UK, and supported by the Medical Research Council with collaborators from the University of Bordeaux, the Universidad del País Vasco and the University of California.

The study showed that CLR01 is able to decrease clusters of the Parkinson’s protein alpha-synuclein forming and prevent the death of human neurons that were made from stem cells.

Researchers tested CLR01 in a mouse model of Parkinson’s that stimulates the formation of protein clusters and mimics the motor symptoms experienced by people with the condition, which can include tremor and slowness of movement. As the mice aged, CLR01 treatment reduced the appearance of motor problems and the formation of toxic protein clusters in the brain. Importantly, the team showed that in much older animals with more progressed Parkinson’s, CLR01 treatment was less effective.

This work shows that using protective therapies early on in Parkinson’s is essential for an effective treatment. These combined results highlight that CLR01 represents a candidate to treat Parkinson’s, and highlights the need for further research in this area.

Lead Researcher Dr Nora Bengoa-Vergniory said: “Future investment to determine the appropriate therapeutic window for these kinds of therapeutic agents is crucial for the success of these and other therapeutic strategies.”

Head of the Oxford Parkinson’s Disease Centre and senior author on the study Professor Richard Wade-Martins said: “This is a very exciting piece of work showing that drug treatments can be developed to unpick toxic protein clusters to save neurons in models of Parkinson’s. Our work is focused on developing new approaches to saving neurons when they start to lose function early on, but before they die later on in the condition. This is a very exciting piece of work showing that drug treatments can be developed to unpick toxic protein clusters to save neurons in models of Parkinson’s. This work should give encouragement to those ultimately hoping for protective therapies.”

Dr Beckie Port, Research Manager at Parkinson’s UK, said: "We desperately need treatments that protect brain cells in Parkinson's. These findings show that this innovative 'molecular tweezer' approach has exciting potential in the lab. We now need to take this therapy forward to test in clinical trials - only then will we know whether it can do the same in people with Parkinson's."

 

The research paper “CLR01 protects dopaminergic neurons in vitro and in mouse models of Parkinson’s disease is published in Nature Communications.

This story has been featured in The Times: "Brain 'tweezers' could treat Parkinson's".

Similar stories

What is the Role of Science in Mental Health?

A new summary report, What science has shown can help young people with anxiety and depression - Identifying and reviewing the 'active ingredients' of effective interventions, from Wellcome has been published. It includes new research from Oxford University, which investigates the knowns and unknowns of SSRI treatment (antidepressant drugs) in young people with depression and anxiety.

New Policy Briefing Addresses Mental Health Effects of the Pandemic on Young People

In the Briefing, a team of researchers at King’s College London and Oxford University highlight the multiple effects that the COVID-19 pandemic has had on children and young people in the UK in their education and daily life, including challenges around social isolation, academic pressures, adjusting to online learning and coping with reopening of schools.

How our dreams changed during the COVID-19 pandemic

This study explored associations between COVID-19 and dream recall frequency, and related social, health, and mental health factors.

New insights into the effect of exposure to dim light in the evening on the biology of the sleep-wake cycle

A new study has revealed more about how exposure to dim light in the evening affects circadian health. The findings emphasise the need to optimise our artificial light exposure if we are to avoid shifting our biological clocks.

Blood lipoprotein levels linked to future risk of amyotrophic lateral sclerosis

Greater understanding of the role of lipoproteins could support screening and efforts to develop treatments.