Clinical Research Office. A partnership between Sheffield Teaching Hospitals NHS Foundation Trust and the University of Sheffield

£2.2m gene therapy boost could unlock cure for one form of motor neurone disease

  • Therapy aims to silence the faulty SOD1 gene known to cause a  specific form of MND
  • Groundbreaking research funded by £2.2 million philanthropic donation
  • Goal is to get the therapy to patients in the clinic as soon as possible

 

SCIENTISTS from the Sheffield Institute for Translational Neuroscience (SITraN) have begun groundbreaking research to develop a gene therapy that, if successful, could cure one form of motor neurone disease.

Motor neurone disease is a rare incurable illness, which leaves sufferers unable to move, walk, talk and eventually breathe. Treatments are very limited, with most patients only expected to live two to five years after diagnosis. Around half will die within 14 months.

Professor Mimoun Azzouz, leading Gene Therapy Scientist and Chair of Translational Neuroscience, said: “This pioneering project has the potential to deliver the first meaningful neuroprotective therapy for motor neurone disease, 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 motor neurone disease cases may be as close as we can get to a cure for motor neurone disease in the near future. Our ultimate goal is to get the gene therapy for SOD1-related motor neurone disease 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 the disease caused by alterations in a gene known as SOD1, but we intend to broaden the approach to other groups of motor neurone disease patients in the future.”

The pioneering research is being funded by a £2.2 million donation from an anonymous donor, and is being jointly led by gene therapy expert Professor Mimoun Azzouz and world-leading motor neurone disease expert Professor Dame Pamela Shaw – the founder of SITraN and a consultant neurologist at Sheffield Teaching Hospitals NHS Foundation Trust.

“Motor neurone disease is considered a rare disease and therefore tends to be overlooked in terms of funding for the development of new therapies,” said Professor Dame Pamela Shaw.

“This emphasises the importance of the extremely generous donation made by a philanthropist based in the USA, for our gene therapy research programme and for our patients with motor neurone disease 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 the disease. We plan submission for regulatory approval by August 2015, for permission to take this therapy to patients in the clinic.”

Researchers at SITraN will now start a large preclinical 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 (SMA) which is a childhood form of motor neurone disease.

The £18m Sheffield Institute for Translational Neuroscience centre brings together 150 international clinicians and scientists working together to fight the disease. It is led by Professor Pamela Shaw, a consultant neurologist at Sheffield Teaching Hospitals NHS Foundation Trust.

For further information visit http://sitran.dept.shef.ac.uk/