UK MND Research Institute: Updates for late 2024

As we move towards the end of 2024, having recently marked the 1st anniversary of the formal launch of the UK MND Research Institute, we bring you 4 brief updates from the Institute and the wider UK research environment. Pieces are starting to fall into place.

We are seeing exciting progress, but with the normal caveats of science. There are no promises, treatments for MND can never come soon enough and we have to be honest in saying that the ‘we’ in this article may not live to see them. This is the brutal truth of our disease.

1. Important and significant financial investment from the USA in the UK Research environment

Investment is vital to ‘cracking’ our disease. The government’s key £50m investment for the Institute, in 2021, started a strategy that we all hope will lead to meaningful breakthroughs for our challenging disease through the formation of a sustainable, innovative research UK research and trial infrastructure.

Building these solid foundations is vital, otherwise failure will follow failure in generation after generation of MND Patients. We need solid evidence based science that leads to a greater understanding of the pathology of our disease. With such a foundation the pipeline of drug candidates can be fed and advance the treatment development process.

It was, therefore, especially welcome news, that UK MND Research Institute member, University College London, was recently, the beneficiary, along with Stanford University in the USA, of a significant £78m funding stream for a newly identified disease treatment target and development of a corresponding gene therapy that may be applicable to many MND patients. For more background on this work please see the following paper. We should all be excited, but realistic, about this project.

Why excited? Because the large funding is from venture capital and put simply, scientists have presented such a compelling case to the investors, and their scientific advisors, that there is true potential in this avenue. Why realistic? Because at the same time, there is a massive amount of work required before potential first human trials.

2- EXPERTS-ALS to start recruiting within weeks

Very soon, the first major patient facing project of the UK MND Research Institute, EXPERTS-ALS, will open its doors. In short, this first of a kind’ drug screening platform’ will rapidly test repurposed (and other) drugs to detect whether they should continue to an essential, but expensive, full trial to test effectiveness.

Will it find winners? We hope so, but one thing it will do is reject losers quickly, in less than 6 months. Hopefully we will start to see less ‘fruitless’ long trials with, to coin a phrase, much ‘better’ shots on goal!

We wrote about EXPERTS-ALS, as patient representatives (PPI), earlier this year in our Behind the scenes post.

Study leaders Professor Chris McDermott (co-director of the UK MND Research Institute) and Professor Martin Turner have just released a short 20 minute video discussing the rationale behind EXPERTS-ALS. It’s an excellent watch! Grab a coffee!

3 – Good News! NICE has agreed in the UK to assess gene therapy for SOD1 MND under appropriate pathway

There was excellent news last week when NICE agreed to reassess tofersen under the HST pathway This means it is far more likely to be fully approved for use under the NHS. UK MND Research Institute co-directors, Professor Ammar Al-Chalabi and Professor Chris McDermott, were instrumental in their many meetings with NICE in achieving this breakthrough and we are all thankful for NICE being so willing to work with the UK MND community. Thank you.

Whilst we await, for what we hope will be, full approval we are thankful to Biogen for continuing to supply the drug free of charge to all eligible UK SOD1 patients.

4 – Finally in this short update, we ‘pause’ our 18 month long efforts to expedite the results of the Mirocals low dose Inteleukin 2 trial

We ‘paused’ campaigning in September (2024) after deciding we were ‘hitting our heads’ against a proverbial brick wall. The results will appear when they will. This very sad saga, is not over yet, and it is one which the UK MND Research community can’t be proud of. Lessons must be, and will be, learnt.

To that end, one of the very best ways we as a community can help prevent such situations developing is for patients to act as representatives (PPI) on research projects. Don’t be afraid, put yourself forward to our charities for example or mention to your neurologist that you might be interested. PPI is a major UK government directive and patient input is vital in both disease research and clinical trial planning.

Of course, we all hope the Mirocals results will be finally released sometime in the new year and that the trial was successful. We will return to the topic at that time. Since ceasing our campaigning, on September 28th, there has been no substantive progress but we will update our Mirocals page when there is a change.

#united2endmnd