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Progress on Developing Gene Therapy for Motor Neurone Disease


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

It is very difficult to find effective treatment for patients with amyotrophic lateral sclerosis (ALS), kind of motor neurone disease which the world-famous scientist Stephen Hawking is fighting with. However, there is now some good news that fundamental progress made from experiments on mice might lead to the breakthrough in the future treatment of such disease.

As Professor Mimoun Azzouz of the University of Sheffield said, ALS/MND is typical of the continuous loss of nerve cells (motor-neurons) that link the brain with the muscles controlling body movements. When the disease becomes serious, patients would be unable to walk, move, eat, talk and even breathe in the end.

Inherited ALS is caused by faulty SOD1 genes. If the gene has default, it will generate the misshapen proteins, thus killing the motor neurones in which they function.

At the moment, a program undertaken by the University of Sheffield is targeted on fixing the genes for prevention of producing the warped proteins .According to Azzouz’s estimation, if doctors could silence the SOD1 gene related with 20% of familial MND cases, they would possibly get easy access to a cure for MND in the near future.

The ultimate goal of Azzouz’s team is to make the gene therapy for SOD1-assocaited MND available in the clinic as soon as possible, because gene therapy is believed to be an innovative technique which has great potential in the treatment of neurological conditions, such as Motor Neurone Disease.

Being funded by a £2.2 anonymous donation, the project would start with preclinical trails immediately. Professor Pam Shaw, the main collaborator of Azzouz said, they were planning to submit for regulatory approval by August 2015, which would permit such therapy to be taken for patients in the clinic. In addition, they are intended to go for the test in gene therapy for Spinal Muscular Atrophy, a childhood onset form of MND.

Although some successes have been made so far, gene therapy is still in its primary stage. More work need to be done in comparison of the tests of enriching the spinal fluid with copper, which are going on well now at Melbourne University.

However, it is hoped that success of Azzouz and Shaw’s work should realize the effective cure rather than the alternative. At present, apart from the two approaches mention-above, there is a third approach aiming to use pluripotent stem cells for  slowing down the disease’s progress in mice.

Source: The University of Sheffield