Field of Science

Gene therapy for MND – the truth behind the headlines

An article published in the Daily Mail, and a number of other news outlets, on 11 July 2014 highlighted how scientists may have discovered a cure for a form of MND, with clinical trials beginning in 2015. Here I report on the science behind the headlines.

The main news behind this article is that the Sheffield Institute for Translational Neuroscience (SITraN)  recently received an anonymous donation of £2.2 million to help translate their MND research from the lab to the clinic. This is a huge amount of money into MND research and this donation will enable the researchers to further our understanding of the disease.
Reporting the news
Although a number of news outlets reported accurately on the news (including the Scotsman), the Daily Mail article received the highest attention highlighting “scientists say that they have found a potential cure for MND”.
But why would two news outlets report the same story so differently? It’s all down to good/bad science communication.
Daily Mail: Have Scientists found a cure for Professor Stephen Hawking’s MND? Clinical trials as early as next year as potential cure is discovered
The Scotsman: Scientists believe they are close to testing a promising treatment for patients struck down with a form of MND
The facts
As a bit of background information, we know that approximately 10% of cases of MND are inherited. The rest of the cases are thought to be caused by a combination of lifestyle, environment and subtle genetic factors.

Inherited MND cases are characterised by a strong family history and the disease is caused directly by a mistake in a specific gene. Of these 10% of cases, 2% are caused by the SOD1 gene (meaning that for every 100 cases of MND, 10 cases are inherited and of these, only 2 are directly caused by the faulty SOD1 gene). To put this into context, approximately 5,000 people are living with MND in the UK, approximately 200 of these have this rare SOD1 inherited form of the disease.

Image taken from
Prof Mimoun Azzouz research at SITraN was reported in a number of news outlets, highlighting how his research is paving the way to a treatment for a rare form of MND.

His research is at a relatively early stage, where he has only just begun investigating the use of a technique known as ‘gene therapy’ in mice affected by the SOD1 inherited form of MND. If the research goes to plan, he will be able to submit a proposal for regulatory approval by August 2015. After approval has been granted, clinical trials will then start. This does not mean ‘clinical trials will start in 2015’ – submitting an application for approval and getting approval, through to starting a clinical trial are very different things that do not all happen at once!

The Scotsman rightly highlighted ‘a form of MND’. However the Daily Mail went along the lines of ‘have scientists found a cure for Professor Stephen Hawking’s MND?’ As you can see, from the Daily Mail article, they’ve used the term ‘cure’. This is dangerous as unless this is the case it can offer false hope to those living with this incurable debilitating disease. Secondly, the Daily Mail article highlights that it is ‘a cure for Professor Stephen Hawking’s disease’. These could wrongly be misinterpreted as a cure for all forms of MND, or even be misinterpreted that Stephen Hawking has this exact form of the disease and this potential treatment is solely for him – big misinterpretations of the science.
As mentioned above, this treatment is firstly being trialled in mice with the SOD1 form of disease, meaning if it makes it to humans it’s only going to be beneficial for people with the SOD1 inherited form of the disease. This is a tiny proportion of total MND cases, therefore using the word ‘cure’ is widely exaggerated. As this treatment hasn’t been tested in humans, there’s no evidence at present to suggest it will be beneficial. Therefore looking at the science, the Daily Mail’s article’s is misleading and creates false hope of a cure, whereas the Scotsman correctly says, ‘scientists believe they are close to testing a promising treatment for a form of MND’, highlighting how amazing this science is but without over exaggerating it.

What is gene therapy?
Gene therapy is a bit like a car company stopping the production of a specific type of car, huh? Well check out my blog on to find out more about gene therapy and cars!
Please note the views expressed in this blog article are entirely my own. Please note that my personal views do not necessarily reflect the views of my employer, academic institute or PhD supervisors.

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