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A gene therapy approach to enhance recovery after stroke

Project details

Dr Lawrence Moon
King's College London
Research area
Brain and spinal cord injury
Funding type
Project grant
Awarded in
September 2017


Better awareness and improved treatment have both contributed to a reduction in mortality from stroke. But many survivors are left with disabilities that limit their ability to live productive, independent lives. Improving stroke recovery is therefore a key clinical and scientific goal.

Dr Moon is developing a new therapy that aims to improve recovery in stroke patients by enhancing the ability of the brain and spinal cord to ‘rewire’ itself.

Following rigorous assessment as part of our competitive project grant round, this project was selected for funding under our ‘acquired brain and spinal cord injury’ theme. The members of our Scientific Advisory Panel felt that the project would make an important contribution to knowledge in an important area, with high unmet need.

Members of the Panel were impressed by the clever idea, which is supported by extensive preliminary data and maps out a clear pathway for translation to human trial.

About stroke

A stroke occurs when blood supply to part of the brain is cut off. This starves the brain of oxygen, with devastating consequences. Stroke is the fourth single largest cause of death in the UK, and a leading cause of disability.  

The number of deaths from stroke is less than half what it was 30 years ago. This is due in part to a reduction in the incidence of strokes, but also to a greater awareness of symptoms - meaning that help is sought sooner - combined with availability of better emergency treatments.

The lower mortality rates mean that more people are surviving stroke than ever before and it is estimated that there are 1.2 million stroke survivors in the UK today.

Over half of all stroke survivors are left with a life-limiting disability. The range of disabilities associated with stroke includes limb weakness, impaired mobility, visual impairments and problems with speech, balance and co-ordination. Fatigue, depression and anxiety are common among survivors.

In addition to the huge personal impact on the lives of stroke survivors, the impact on society is enormous. The economic burden of stroke in the UK is estimated at £9 billion a year - including health and social care costs, informal care, productivity losses and benefit payments. Two-thirds of working age survivors are unable to return to work.

Improving stroke recovery to enable survivors to live independent, productive lives is a therefore a key goal.

Read more: Stroke

Enhancing recovery after stroke

The brain and spinal cord have a remarkable ability to ‘rewire’ themselves after injury. This rewiring process – called neuroplasticity - can take place over many months and years and helps to explain how, with the help of rehabilitation, people can recover functions that were initially lost as a result of injury.

Rehabilitation can be slow and frustrating, however, and often stretches the resources of healthcare systems, which struggle to deliver enough rehabilitation in the timeframe required.

Considerable research effort has been devoted to finding ways to enhance neuroplasticity in order to facilitate faster, more complete recovery from brain injury.

Dr Moon and colleagues are developing a new therapy designed to enhance neuroplasticity in stroke patients. They have already shown how their therapy improves walking in adult and elderly rats after stroke and brainstem injury, and they are now refining the approach. The therapy involves delivery of a growth factor (Neurotrophin-3) that helps rewire brain and spinal circuits after stroke.

They are carrying out essential testing in rats, as a precursor to moving to human clinical trial. They have developed so-called “RatBots”, which provide automated, in-cage delivery of sugar pellets to promote rehabilitation of grasping in the rats.

They will assess the consequences of treatment in rats, using methods that could be applied to humans in a clinical trial.

The team’s longer-term objective is to run a Phase I trial of this therapy in human patients after stroke, and this project will pave the way for such a trial.


The impact of stroke is enormous – at both an individual and a societal level.

Improving stroke recovery is a crucial clinical and scientific goal but our healthcare systems struggle to deliver enough rehabilitation in the timeframe required. A treatment that enhances recovery could benefit hundreds of thousands of stroke survivors in the UK alone. And whilst this project focuses on stroke patients, the treatment will also have applicability to those affected by other forms of neurological injury.

This is a proof-of-concept experiment, supported by extensive preliminary data, using rats to test a new therapy for stroke.

It has clinical promise because it is based on a treatment that is already known to be safe in humans, and the administration of which is feasible in a timeframe that is relevant for stroke.

If the therapy, combined with intensive rehabilitation, leads to a large enough benefit in rats, the team will seek funding to take the therapy forward to human trial.  

This is a project with clear aims and whose completion could be very significant in helping to treat a significant brain problem,

- External reviewer.

Related projects

Acquired brain and spinal cord injury (including stroke) is one of our current research priorities, reflecting the large unmet need in this area. Our aim is to fund research to advance understanding of how to promote repair of the brain and spinal cord following injury.

Read about our other research projects under this theme:

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