As an interested party, ie a patient, I would like to give a short onein300 commentary on the current ongoing government Motor Neurone Disease research funding campaign (#United2EndMND) here in the UK.

A unique collaboration between the MND Association, MND Scotland, My Name’5 Doddie Foundation, researchers and patients, #United2EndMND is a drive for a critical and timely new government investment (ie over and above current spending) for clinical research infrastructure for MND.

A fresh injection of £50m over 5 years would be a truly game changing figure.

I know, however, that some reading today might possibly say that such an amount isn’t really ‘all that’ much and is it really enough?

But, consider this……

£50m would essentially represent about a 300% increase in yearly UK Government targeted MND research investment! Wow!

So what, exactly, is this research infrastructure, Lee?

#United2EndMND is, at its core, focused on laying strong, firm and maintainable foundations to discover, screen and trial new treatments to enable the rapid acceleration towards a world free from MND.

It’s time for an ‘analogy’ to really set the scene.

The epic TeamGB London 2012 Olympic achievements were not the product of chance, far from it….

London 2012 – Olympics Opening Ceremony – Nick J Webb, CC BY 2.0, via Wikimedia Commons

The current state of MND research in the UK could be likened to Great Britain’s past Olympic sporting achievements before the groundbreaking 2012 Olympics in London.

Although always blessed with world class athletes, our nation was consistently, at best, a ‘middling’ player in terms of the sheer medals tally and always outside the top 10 in the modern era.

But after winning the bid to host the 2012 Olympics back in 2005, the investment and leadership over the subsequent buildup, and in the application preparation, enabled the gold rush that propelled GB into the top 3 in the medals table in 2012. Great Britain went on not only to maintain this level, but achieved 2nd place in 2016! Our little, tiny nation had roared.

This is what a timely investment can realise.

The existing MND research ecosystem is very comparable. We have world leading scientists making innovative discoveries. They receive a trickle of funding, start a project, but are then not able to maintain the momentum through lack of continued funding. Is this a reason why industry investment might just choose to go elsewhere for clinical trials in some instances?

#United2EndMND is a mission to break this log jam, through the introduction of an MND Research Institute.

The MND Research Institute (MNDRI) Vision

The MNDRI has bold aims, somewhat mirroring those of the world class training infrastructures that leading GB athletes use daily underpinned with vibrant leadership, governance and strategy.

So here are a few items of note about MND and the MNDRI that I hope will give you insight and help our community if you are ever in discussion with friends, family, MPs, influential people etc.

MND is a brutal disease that needs to be tamed

We have, in all but name, a continual epidemic. MND is a relentless killer that has consistently taken, yearly, significantly more lives than HIV in the UK, even at the height of the horrific and devastating AIDS crisis. That’s quite a surprising statistic, isn’t it?

MND will now go on to kill about 200,000 of the current UK living population unless we start to develop and bring to fruition effective treatments. MND is currently, an ‘unavoidable’ disease and almost 100% fatal.

Targeted research – government funding is low

Despite significant amounts being spent on neurological disease research, the targeted funding for realising a treatment for MND from the UK Government is under £4m a year.

Why are we campaigning now?

Even with such funding issues, British scientists are closing in on treatments for some members of our MND family. Our researchers are true world leaders having contributed a vast amount to global MND discoveries, punching way above our weight.

Genetics holds the secret to unlocking the disease and will stimulate the momentum financially and technically to realise treatments for MND.

The first most likely major breakthrough will be with a genetic form of MND. Once proven, alongside continuing searches for drugs (including further gene therapies, even for sporadic forms (at least 2 in trials now)) industry investment will probably escalate. However, industry need to have a real incentive to bring hard cash to our shores.

That incentive will be the MNDRI.

Bringing efficient trials to the MND population is a major building block of the MNDRI, with new technologies and analytical techniques to measure and predict individual disease progression more precisely and target potential treatments for our highly varied disease.

Some of you may have heard of the existing MND-SMART trials. I have seen questions on social media recently asking just how does the MNDRI fit with these?

MND-SMART is an ‘adaptive’ trial platform. Designed to a be rapid start/stop treatment trial platform it is an essential element to accelerating the arrival of treatments, and an important component of the MNDRI. It is a vital and pre-requisite step forward. However, it is but one piece of the jigsaw. Drugs to be put through such a platform will be continually refined based on laser focused pre-clinical studies and analysis.

The MNDRI aims to fully integrate a streamlined system starting from therapy target identification, through pre-clinical screening of treatments for identified targets, on to selecting appropriate patients for trials and finally running on a platform trial system, including MND-SMART and others, such as TRICALS.

It is this lifecycle of development from lab to patient back to lab that currently is typically quite ad-hoc globally with programs being set up, ripped apart and having to be recreated continually for different treatment prospects.

Pharmaceutical companies spend vast amounts on trials, but can struggle with recruitment and logistics. The availability of a trial population, NHS integrated, with an on-demand platform in the UK will attract industry. If the UK Government doesn’t invest, companies may take the path of least resistance and go elsewhere. The #United2EndMND campaign has strong support from the leading pharmaceutical companies.

Targeted – the essence of a developing strategy

It is often said that research into one neurodegenerative disease will help research in another. Although basic science will benefit, a lot of this is probably wishful thinking in my opinion. With MND the science is further down the line in disease understanding, perhaps, than diseases such as Alzheimer’s and other dementias. Several disease targets have been discovered, and to accelerate MND disease research focus has to be placed on these rather than general research. To add to this, because MND diagnostic accuracy is higher and changes in the disease process are easier to measure, the benefits from MND research are more likely to beneficial to the more widespread neurodegenerative diseases.

It is important to note, that, to date that the ONLY neuro-degenerative disease altering drug approved anywhere (ie slowing the course of any such disease, including the dementias) is Riluzole for MND! And we know how marginal that is.

How long will this take?

As patients we are all unsurprisingly seeking a time frame for when we as individuals might see the benefits of research. This is undoubtably a question which clinicians and researchers must dread and fear.

It will take time, and several years is still the realistic answer, but leading scientists are highly confident that some forms of our disease will have treatments within the not too distant future.

The MNDRI will set the infrastructure for truly accelerating treatments and set the bar for generations to come.

It won’t materialise overnight and success is not guaranteed. Just like the Olympic dream of 2012, it might not happen for all, but the legacy will be forever.

Finally, if there is one sentence that describes #United2EndMND for me, it is

“Targeted funding for targeted research equals success”

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