Progress on Gene Therapy for Motor Neurone Disease

The Sheffield Institute for Translational Neuroscience. Gene therapy for motor neurone disease may be on the way

For people with amyotrophic lateral sclerosis (ALS), the form of motor neurone disease affecting Stephen Hawking, good news is rare. Now, however, hot on the heels of treatment success in mice comes an announcement of progress at a more fundamental level.

“ALS/MND is characterized by the progressive loss of nerve cells (motor-neurons) that connect the brain with the muscles to control movements. As the disease progresses, patients may lose the ability to walk, move, eat, talk and finally breathe,”  says Professor Mimoun Azzouz of the University of Sheffield

Inherited ALS is a result of faulty SOD1 genes. When the gene is defective it produces proteins that become misshapen and kill the motor neurones in which they function. A program at the University of Sheffield aims to fix the genes to prevent the production of the warped proteins.

"Silencing the SOD1 gene that is associated with 20% of familial MND cases may be as close as we can get to a cure for MND in the near future,” Azzouz says. “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.”

The project has been funded by a £2.2 anonymous donation, allowing preclinical trails to start immediately. Professor Pam Shaw, who is leading the team jointly with Azzouz says, “We plan submission for regulatory approval by August 2015, for permission to take this therapy to patients in the clinic." They are also preparing for a trial of gene therapy for Spinal Muscular Atrophy, a childhood onset form of MND.

Despite some successes, gene therapy is still in its infancy, so progress may prove slower than the approach being tested at Melbourne University of enriching the spinal fluid with copper. However, if Azzouz and Shaw's work succeeds it should provide something closer to a cure than the alternative. Meanwhile a third approach, using pluripotent stem cells to slow the disease's progress in mice, has also been announced in the last month.

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