Scientists claim to have discovered a possible cure for motor neurone disease (MND), the condition which affects Professor Stephen Hawking, according to a report in the Daily Mail.
Researchers from the Sheffield Institute for Translational Neuroscience say the treatment could be undergoing clinical trials as early as next year. They say that this ground-breaking research could lead to a cure for at least one form of the disease. MND is known to affect over 5,000 people in the UK, the most famous sufferer being Professor Hawking.
Professor Mimoun Azzouz, Chair of Translational Neuroscience and a leading Gene Therapy Scientist, said:
“Quite simply this is a devastating disease, patients suffering from motor neurone disease can die within one or two years in the most severe cases, and lose all ability to use muscles, including arms and legs. It can prevent them from having a normal life and there is no treatment at all for this.
“Some of the cases are genetic, caused by a faulty gene called called SOD1. We are devising a strategy to take out the faulty gene.
“The breakthrough, if this a success, will be huge because there is no treatment for this syndrome. Because it is genetic, if patients can be screened for the gene, because of family history, then we can intervene even before the symptoms appear and rescue them. It can be a cure.”
He added, “We plan submission for regulatory approval by August 2015, for permission to take this therapy to patients in the clinic. It will be in clinics as a clinical trial at first, then if that is a success we would look to make it available for patients after that.”
Observers hope that his confidence is justified since, until now, treatments and drug-trials carried out internationally have all failed to halt the progression of the disease. Only one drug, Riluzole, has been shown to prolong life for three to six months, and is not effective for everyone.
Prof Azzouz is confident that this research will be successful. He explained his optimism like this:
“Because we are targeting the causative gene, based on the previous studies that we have done, there’s a very good chance that this can be a success.
“We are in an advanced program looking at MND in childhood and in babies, and it’s cause is not because of a faulty gene but it is in fact totally absent. In our program we are using a virus carrier to attempt to restore the gene back.
“For example, in a mouse model the mice that had the disease would survive two weeks maximum from birth. But when we treated these mice we seen that they survived for nearly 400 days, so you can see the strength of this strategy.”
But Stephen Hawking is not a mouse, so for him, the sooner the clinical trials are completed the better. Hopefully he will still be around to benefit from the results.