Scientists in Yorkshire have launched ground-breaking research to develop a gene therapy to cure a devastating neurological disorder.
The work by leading experts at Sheffield University aims to silence a faulty gene which triggers an inherited form of motor neurone disease (MND).
The world-leading research is being funded thanks to an anonymous £2.2 million donation from the United States.
Specialists describe the project as a “beacon of hope” for patients and plan to seek approval next summer from regulators to take the therapy to patients.
Until now, all available treatments and drug trials around the world have failed to effectively halt the inevitable progression of the illness which affects more than 5,000 patients in the UK. Only one drug has improved survival in patients but it prolongs life for just three to six months and may not be effective for all.
Now researchers from the Sheffield Institute for Translational Neuroscience (SITraN) are focusing their efforts on a faulty gene SOD1.
Leading gene therapy scientist Prof Mimoun Azzouz, who is co-leading the work, said: “This pioneering project has the potential to deliver the first meaningful neuroprotective therapy for MND, offering a real beacon of hope for patients and their families living with this cruel disease.
“Silencing the SOD1 gene that is associated with 20 per cent of familial MND cases may be as close as we can get to a cure for MND in the near future.
“Our ultimate goal is to get the gene therapy for SOD1-related MND into the clinic as soon as possible.
“Gene therapy is regarded as an innovative technique with huge potential for the treatment of neurological conditions including motor neurone disease.
“We plan to use the gene therapy initially in an inherited form of MND caused by alterations in a gene known as SOD1, but we intend to broaden the approach to other groups of MND patients in the future.”
Project joint lead and world-leading MND expert Prof Dame Pam Shaw, said: “MND is considered a rare disease and therefore tends to be overlooked in terms of funding for the development of new therapies.
“This emphasises the importance of the extremely generous donation made by a philanthropist based in the United States, for our gene therapy research programme and for our patients with MND who don’t have time to wait.
“The experience that we have accumulated with our gene therapy and clinical trials programmes at SITraN is crucial to develop this new therapy for patients with MND.”
Researchers will start a large pre-clinical study before the first-in-human clinical trial.
The team are also currently preparing for the first ever UK gene therapy trial in humans for spinal muscular atrophy which is a childhood form of MND.
MND leads to the progressive loss of nerve cells, or motor neurons, that connect the brain with the muscles to control movements. Messages gradually stop reaching muscles leading to wasting and weakness. As the disease progresses, patients may lose the ability to walk, move, eat, talk and finally breathe.
Around seven in every 100,000 people have the condition which tends to affect twice as many men as women, mainly aged 50-70. Between five-10 per cent have inherited forms of the illness.