New therapy may be key in neurone disease battle

Professor Stephen Hawking's achievements have kept MND in the public eye. Picture: Getty
Professor Stephen Hawking's achievements have kept MND in the public eye. Picture: Getty
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SCIENTISTS believe they are close to testing a promising treatment for patients struck down with a form of motor neurone disease (MND).

Researchers in the UK have developed a therapy to target a gene linked to some cases of the devastating condition which, they believe, could be as close as they can get to a cure for such patients.

They hope to start tests of the gene therapy next year, before expanding research to see if it could help wider MND sufferers and potentially be used to stop the disease developing in those where a genetic link is found.

The disease, which affects around 5,000 people in the UK, causes parts of the nervous system to become damaged, leading to weakness, muscle wasting and loss of the ability to walk, speak or breathe.

The disease has affected public figures including scientist Professor Stephen Hawking, Celtic footballer Jimmy Johnstone and Scottish theatre producer David MacLennan, who died last month.

A team from Sheffield University is developing a therapy which targets a gene known as SOD1. It is linked to some cases of MND where there is a family history – familial MND.

Around 5 per cent of all MND cases are familial, of which about 20 per cent have links to the SOD1 gene. This potentially means between 1 and 2 per cent of the 5,000 cases in the UK at any one time could benefit from the treatment being developed in the study.

Until now, the only drug that has demonstrated a “survival benefit” in MND patients is Riluzole, which prolongs life for three to six months and may not be effective in all patients.

Professor Mimoun Azzouz, leading gene therapy scientist and chair of translational neuroscience at Sheffield, said their project had the potential to deliver the first meaningful therapy for MND which offered “a real beacon of hope for patients and their families living with this cruel disease”.

The gene therapy would work by targeting and switching off the SOD1 in these patients.

“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 in the near future,” Prof Azzouz said.

“Our goal is to get the gene therapy for SOD1-related MND into the clinic as soon as possible.”

Prof Azzouz said while the initial focus of the research, being funded with a £2.2 million donation from an anonymous donor, was on the SOD1 gene, they hoped the research would eventually be widened to cover other forms of the disease and help a larger number of patients. A spokesman for charity MND Scotland said: “Even though this gene is responsible for MND in only one or two out of every hundred cases, it is promising to hear of these results from translational research.

“The possibilities raised by this work should be treated cautiously at the moment as it has not yet been tested in humans.

“Blocking the mutated gene will not give recovery to people whose nervous system has been already damaged, but if the treatment does translate over it should either prevent or slow down further deterioration and extend lives.”


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