Time to get MND-SMART with breakthrough trials

Annually, 1,100 people in the UK are diagnosed with motor neurone disease. For decades, the degenerative disease has, on the surface, not seen any movement towards a cure. In 2020, all this looked set to change, Professor Ammar Al-Chalabi speaks to Lorne Gillies about the future of MND research.

There is a one in 300 chance of developing motor neurone disease (MND), which can affect people in different ways.

MND is a group of diseases that affect the nerves, called motor neurones, in the brain and spinal cord that tell your muscles what to do.

Affecting adults of any age, MND affects 5,000 adults in the UK at any one time. At present, there is no known cure for MND with life expectancy estimated to be three years from initial signs of symptoms.

Professor Ammar Al-Chalabi

However, there is no hard and fast definitive when it comes to MND with the disease progressing rapidly or slowly. Some forms of MND can be treated, in a limited capacity, by a drug called riluzole, which has been in distribution for over 25 years with no new breakthroughs; until now.

TRIALS

“Testing of medicines has largely failed because our understanding of the disease hasn’t been great,” explains Professor Ammar Al-Chalabi from King’s College London, and also a member of My Name’5 Doddie Foundation’s Scientific Advisory Board.

“We’ve not had the technology to understand the disease until recently.

“Now, we’ve got multiple trials and a lot happening. Right now, I have eight trials being set up and another two or three in the early enquiry stages; that’s far more that we’ve ever had before.

“If you’re testing 10 drugs instead of one a year, then you’re doing research 10 times as fast. Also, we’re shooting closer and closer to the target: our understanding of the disease is getting better, so we are getting better at aiming,” enthuses Prof. Al-Chalabi.

One such trial working to speed up the time it takes to find medicines targeted to slow, stop, or reverse the progression of MND is MND-SMART.

MND-SMART

A pioneering clinical drugs trial will see hundreds of people living with MND across the UK take part in the testing of potential treatments.

It is an incredibly exciting time, with MND-SMART working as a multi-arm trial, meaning that unlike typical clinical trials that look at testing single treatments, MND-SMART will take different treatments compared to a single group taking a dummy drug.

Ultimately, with MND-SMART more people are likely to receive active treatment.

Prof. Al-Chalabi explains: “MND-SMARTwhich originated in Edinburgh – is a really innovative trial. It is a platform trial where you study multiple drugs at the same time, meaning you can get your answer much quicker to find out what works and what doesn’t.

“There is another trial underway called TRICALS, which is part of a European, and now, international collaboration where we do trials in a faster way,” Prof. Al-Chalabi continues.

“It is now a platform trial as well, where you test several agents at the same time. We’re also doing lots of things in association with the trials, so even if the drug doesn’t benefit people with MND we still learn a lot about the condition and why the trial didn’t work so we can do the next trial faster and better.”

PROGRESSION

As well as clinical trials there are other types of clinical research. For people living with MND they can get involved with clinical trials, however, the wider general public can participate in clinical research to provide wider information and understanding of genes and how MND works, promoting even more knowledge of the causes of MND and potential life-altering medication.

Prof. Al-Chalabi continues: “We need someone who doesn’t have MND to compare their gene variations, or what exposures a person has had in their life, for example, are they sporty or not.”

Similarly, advancements are being made when it comes to precision medicine: where a drug is targeted directly to a sub group of people, which could highlight a lot more about why MND is happening.

“For a precision medicine approach, we are definitely coming close to a breakthrough with gene therapy, it does look like those may well slow it down significantly or potentially stop it,” adds Prof. Al-Chalabi.

The more information the greater the chance of working towards a cure, for all conditions, but MND has made remarkable progress since 1994, when the last drug for MND was discovered.

Faxing results in days gone by has elevated to the swift response of an email, technology advancements are not to be ignored. But, the importance still stands around the number of people participating in trials.

“If I’m trying to identify a cause of MND but I’ve only got 50 people to study, it’s much more difficult compared to if I had 50,000 people to study, or even just 5,000. So, sharing your information with other researchers is vital,” continues Prof. Al- Chalabi.

From sharing research to raising awareness from sporting personalities affected by MND, including Doddie Weir, Rob Burrow, to Stephen Darby and the late, Fernando Ricksen: knowledge of MND is reaching crescendo.

“The more people that know about MND, the more likely people will come forward to participate in trials and also people who are not affected, because we also need people who don’t have MND; we need the general public to be involved,” emphasises Prof. Al-Chalabi.

“People are much more likely to hear about MS but that is because it is not likely to shorten your life, whereas MND does, so there are fewer people around with it – even though you have the same chance of developing either condition.”

With more research and results from ongoing trials, the future of MND medication is certainly looking brighter.

What will 2021 have in store?

BREAKTHROUGH

It appears, 2021 has provided a flurry of excitement around MND after researchers in Edinburgh became one step closer to reversing the damage caused by MND.

On 18 January 2021, scientists at the Euan MacDonald Centre for MND research discovered that drugs approved for other conditions could be beneficial for people living with MND.

The research found that the damage to nerve cells caused by MND could be repaired by improving the energy levels in mitochondria – the power supply to the motor neurons. 

They discovered in human stem cell models of MND, the axon – the long part of the motor neuron cell that connects to the muscle – was shorter than in healthy cells. And the movement of the mitochondria, which travel up and down the axons, was impaired.

At Enable, we will continue to follow the research into MND and provide updates. Keep in touch by following us on social media, TwitterFacebook or Instagram.