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The Foundation attends top meeting of international MND experts

03 August 2023

In July, we attended the meeting of the European Network for a Cure for ALS (ENCALS). The meeting, held in Barcelona, was an opportunity for academics, clinicians, industry, healthcare professionals and funders, all working in the ALS field, to share their research, gain expert feedback and scope out new opportunities for collaboration. We were represented by Olivia Bird, Research Programme Manager, and you can read more about some of her highlights from the meeting in this article.

The meeting was opened by Juan Carlos Unzué, a Spanish football manager and former player, now living with MND. His stoicism and bravery were clear to see and reminded us very much of our founder, who also faced his toughest fight after the end of his professional sports career.


Dr Neil Schneider, Director of the Eleanor and Lou Gehrig ALS Center at Columbia University Irving Medical Center, opened the Therapeutics session with an update on the FUSION trial, a phase 3 trial that is testing an antisense oligonucleotide (ASO) in people living with MND. ION363 is designed to reduce the level of the protein, Fused in Sarcoma (FUS), that is mutated in up to 5% of people living with MND. Typical onset of this form of MND is during childhood or early adulthood. The room fell silent when videos of a patient walking up the stairs following treatment were played in comparison to the video in which the same patient was unable to walk up the stairs 18 months prior. The trial, sponsored by Ionis Pharmaceuticals, is now recruiting at 5 sites across Europe, and we’ll be keeping a close eye on its progress.

Debate - Early to Market or Definitive Evidence?

One of the most highly anticipated sessions of the meeting was a debate on whether new treatments should be made available to people living with MND before regulatory approvals are in place, or whether we should be seeking definitive evidence of a treatment’s efficacy before seeking regulatory approval. The debate, moderated by Professor Leonard van den Berg, was led by Dr Jinsy Andrews, Director of Neuromuscular Clinical Trials at Columbia University (Early to Market), and Chris McDermott, Professor of Translational Neurology at the University of Sheffield (Definitive Evidence). Following the opening statements, the audience voted for which side they were on, with nearly 50% of the audience voting for Early to Market and nearly 40% voting for Definitive Evidence.

Dr Andrews went on to argue we should be comfortable with some residual uncertainty after a clinical trial, and that, although we may not be completely confident in the results today, we may be in the future. To counter, Professor McDermott used the example of dexpramipexole; the phase 2 trial showed promising results while the phase 3 failed to show any benefit at all to people living with MND. He argued that allowing access to the drug before the results of the phase 3 trial would have given false hope to people already living with such a devastating disease.

The session ended with a final poll, and although ‘Definitive Evidence’ took the win, it was clear the community remains very much divided on the topic.

Both Dr Andrews and Professor McDermott agreed there was a subtle lack of harmonisation in regulatory processes across the globe and that a more joined up approach to regulatory approval would ease the pressures faced by researchers to make new treatments available to all people living with MND, no matter where they live. Although the consensus shifted towards ‘Definitive Evidence’, the question remains, what level of evidence will we settle for to allow people living with MND access to new treatments?

We expect this issue to remain at the forefront of the minds of the MND community as more potential drug candidates make their way through the clinical trial and regulatory phases, and we will continue to monitor developments and share the latest news with our supporters.


In addition to hearing about the latest updates in the research field, conferences are a great opportunity to network with others in the community and develop new collaborations. At this meeting, we had the opportunity to talk with representatives from MND Association, Target ALS, FightMND and the International Alliance of ALS/MND Associations, opening new opportunities for working together and we look forward to developing these relationships further over the coming months.

We were pleased to see research projects we have funded in Oxford, Edinburgh and Sussex being presented during the poster sessions. Our researchers shared their work with experts from across the MND research community and we discussed how their progress would have a real impact on people living with MND.

We look forward to attending the next ENCALS meeting in Sweden in 2024.

The Foundation attends top meeting of international MND experts
The Foundation attends top meeting of international MND experts

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