An innovative gene therapy programme has been launched that could lead to novel treatments for neurodegenerative diseases.
The research is being carried out by scientists at the University of Sheffield’s Institute for Translational Neuroscience (SITraN), with the Cell and Gene Therapy Catapult, after being awarded £513,141 from LifeArc and the Motor Neurone Disease (MND) Association.
The study will test the feasibility and efficiency of a gene therapy programme to treat motor neurone disease (MND) and frontotemporal dementia (FTD) patients with underlying mutations in the C9orf72 causative gene.
The researchers hope if it is successful, it could lead ground-breaking research into future clinical trials.
Dr Guillaume Hautbergue, head of the RNA Biology Laboratory, who is leading the study with Prof Mimoun Azzouz and Prof Dame Pamela Shaw, said: “This is a completely new approach to tackling the most common type of motor neurone disease.
“No one has yet attempted to prevent these repeated sequences of RNA from leaving the cell’s nucleus and it opens up new areas of investigation for gene therapy. It is an exciting time for biomedical research and the development of novel treatments for incurable diseases such as MND.”
In an early stage study, published in Nature Communications in 2017, Dr Hautbergue and his research team identified why the repeated RNA sequences can leave the cell’s nucleus to cause cell death.
They discovered the SRSF1 protein, which binds to the pathological repeated RNA molecules and transports them out of the cell centre, overrides the gatekeeping machinery within the nucleus by opening a back door.
They went on to show that reducing the SRSF1 protein or altering its makeup so that it is unable to interact with the cell’s export machinery can reduce the amount of rogue RNA molecules escaping into the cell’s cytoplasm.
Matthew Durdy, chief executive officer of the Cell and Gene Therapy Catapult, said: “The progress of this scientific breakthrough from the University of Sheffield and the support for this programme by leading investors and charities such as LifeArc and the MND Association, is testament to the UK research excellence and favourable ecosystem for cell and gene therapies.
“We welcome the opportunity to be part of this programme and look forward to working with the University of Sheffield to help advance their innovative gene therapy technology into the clinics.”
Dr Brian Dickie, director of research development at MND Association, added: “Gene therapy for neurodegenerative diseases, such as MND, has had its ups and downs over the past two decades, but the field is now enjoying a renaissance. There is a clear route to clinical trials if the outcomes of this new study are positive.”
Dr Melanie Lee CBE, chief executive officer of LifeArc, a self-funded medical research charity said: “This gene therapy approach, focused on preventing motor neurone toxicity, is the latest in a series of initiatives we are proud to be part of to drive research into practical treatments for this fatal and brutal condition.”
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