Reflecting on the 34th International Symposium on MND

18 January 2024

The 34th International Symposium on ALS/MND was held in Basel, Switzerland, over 3 days in December 2023. It brought together over 1600 delegates from around the world, making it one of the largest MND research gatherings to date.

 This Symposium was a hub for groundbreaking updates, covering a wide range of topics from genetic research to advanced clinical management. My Name'5 Doddie Foundation was represented by Jessica Lee, Director of Research, and Giacomo Bertuzzi, Research Programme Manager. In the upcoming weeks, we look forward to providing in-depth insights on critical developments shared at the Symposium. Today, we’re sharing some of our highlights.

Neurofilament breakthrough

The Healey International Prize for Innovation in ALS was awarded to a team of international experts, including our Scientific Advisors, Professor Andrea Malaspina (UCL) and Professor Martin Turner (Oxford). This achievement highlights their important work in establishing the neurofilament protein as a biomarker of MND. Neurofilaments (NF) can now be detected and measured in blood and spinal fluid, enabling monitoring of disease progression. Notably, the FDA approval of tofersen, targeting MND with SOD1 mutations (SOD1-MND), was based on a reduction in NF levels, underscoring their value as treatment response biomarkers. 

To further understand the role of neurofilaments in MND and their potential as biomarkers and therapeutic targets, we are currently funding more research led by Professor Andrea Malaspina, which you can read about here.

Advancements in FUS-gene therapy

The Drs. Ayeez and Shelena Lalji & Family ALS Endowed Prize for Innovative Healing was awarded to Dr. Neil Schneider and Professor Robert Brown, along with their team, for their pioneering gene therapy work on the FUS gene. Mutations in the FUS gene cause a rare, aggressive form of MND that often affects younger people. Their approach, similar to that of tofersen for SOD1-MND, has demonstrated promising results in slowing down and even reversing the progression of this challenging condition, highlighting the vital role of continuous innovation in MND research.

In a moving segment of the Symposium, Ana, who benefited from this gene therapy, shared her inspiring story. Once ventilator-dependent and frail, Ana narrated her journey of regaining strength and walking with increasing independence, eventually freeing herself from ventilator reliance. To learn more about Ana's remarkable journey and the effects of her treatment, watch this short movie.

Ana's case, along with 18 others, played a crucial role in propelling the gene therapy into a phase 3 clinical trial which is currently ongoing. Her account, filled with resilience and gratitude, gave the audience a hopeful perspective on the importance of relentless research and advancement. It underscored the power of scientific collaboration and the meaningful progress that can be achieved. Ana's experience and the dedication of the research team served as a potent reminder that every advancement, no matter the scale, is a step forward in the fight against MND. 

The Symposium provided an opportunity to unveil and delve deeper into forward-thinking therapeutic approaches, including those championed by scientists supported by the Foundation: 

• Professor Pietro Fratta's TDP Reg system: Professor Fratta introduced an innovative approach to treat MND, known as the TDP Reg system. This system focuses on a protein called TDP-43. In around 97% of people living with MND, TDP-43 is mislocalised from its usual place in the nucleus, into the cell's cytoplasm. The TDP Reg system is like a smart detector that specifically targets cells where this mislocalisation happens. It delivers special gene therapies to these affected cells, acting only where it's needed. This approach could be safer than other treatments that affect the entire nervous system, avoiding potential side effects and improving how gene therapies for MND are delivered.

To find out more about TDP-43, check out our infographic, and to find out about other research from Professor Fratta read about the project we are funding here.

• Professor Chris Shaw's gene therapy: Professor Shaw shared his pioneering research in gene therapy, a collaborative effort that included significant contributions from Professor Steven Gill and their team. Their innovative method to deliver gene therapy focuses on the thalamus, a central brain region, utilising its extensive connections to target various brain areas. This technique, developed with the initial support of My Name'5 Doddie Foundation, overcomes key challenges in delivering treatments to the central nervous system. It represents a potentially significant advancement in neurological care, highlighting the unique potential of gene therapy through precise targeting within the brain.

To find out more about Professor Shaw and his team's work, read our recent post about the project we’re funding.

The Symposium was a great opportunity for us to meet up with our international colleagues, old and new, and benefit from the collective knowledge and emerging strategies being developed in MND research.

We extend our thanks to our friends at the MND Association for hosting an excellent conference. Their commitment to organising this event is instrumental in uniting the global MND community.

Stay tuned for more in-depth updates from the Symposium in the weeks to come!