This blog is part of our Symposium Blogathon series – where we are counting down to the 33rd International Symposium. Numbers in bold blue type correspond to the code in the abstract book. Click on the number to be redirected to the full abstract.
Every year, one of the highlights of the Symposium is our plenary speakers. This year we have 15 ALS/MND research and clinical care experts who will be presenting overviews of their research.
To give you a teaser of what is to come, we will be taking a closer look at each of our plenary speakers. This is the second of two blogs about our plenary speakers, where we explore the topics, they will be presenting and find out a little bit more about the speakers. You can read more about the rest of the plenary speakers in part 1 here.
Day 3, Session 7: RNA and Protein Processing
Professor Heather Durham, from the Montreal Neurological Institute at McGill University, and her team investigate the relationship between biological mechanisms of ALS/MND and the properties of motor neurones. This work hopes to further understand why motor neurones are vulnerable in ALS/MND which will help in identifying potential therapies that could protect the neurones.
Proteins are vitally important to the function and structure of all cells in our bodies. In ALS/MND there can be problems with proteins within cells, such as the proteins being made and folded incorrectly or by sticking together to forming harmful clumps within nerve cells. When protein are made in cells a group of proteins called ‘chaperones’ work to make sure that the proteins are built correctly. In times of stress the amount of active chaperone proteins increases to stabilise proteins inside cells. One potential therapeutic strategy for ALS/MND may be to boost the chaperoning capacity, within cells, which would provide additional support for the correct folding of proteins. In her talk ‘Protein chaperones as a therapeutic target for ALS’ (C18), Professor Durham will talk about the methods of boosting chaperoning capacity by inducing a particular group of proteins, called heat shock proteins and further discuss other factors which impact the maintenance of proteins.
Professor Eran Hornstein is the founding Chair of the Department of Molecular Neuroscience at Weizmann Institute of Science, Israel. He is also the inaugural direction of the Andea L. and Lawrence A. Wolfe Family Centre for Research on Neuroimmunology and Neuromodulation. Professor Hornstein and his team use computational and mathematical analysis to model complex biological process to explore the biological mechanisms of ALS/MND.
ALS/MND is a complex disease with many different biological pathways associated with the disease. Understanding these pathways, how they work and why they go wrong in ALS/MND is key to helping to design and develop potential new therapies for ALS/MND. One particular group of molecules, known as microRNAs, are involved in the control of essential pathways that are vital to the function of motor neurones. In his talk ‘microRNA dysregulation in motor neuron disease’ (C19) he will discuss the importance of microRNAs, the biological pathways they are involved in and how understanding how they contribute to different pathways in the body could lead to new therapeutic targets.
Day 4, Session 8: Neuroinflammation
ALS/MND is considered a multifactorial disease, meaning many different factors are known to contribute to its onset and progression. One of these factors is neuroinflammation which is characterised by an increase in the immune response within the central nervous system. This increase in inflammation is thought to contribute to motor neurone damage in the disease. Inflammation within ALS/MND is characterised as the chronic activation of glial cells, such as microglia, changes in T Cell activation patterns and immune system activation.
Dr Jasna Kriz, from the Universite Laval in Canada, and her team focus on the role of microglia and immune response within Brain disorders. Microglia support the motor neurones, and whilst they were originally thought of as merely the ‘glue’ of the nervous system, it is now thought that they play a role in the disease. In her talk titled ‘Modelling neuroinflammation in ALS’ (C21) Dr Krix will discuss recent evidence which suggests that ALS/MND microglia cells lose their protective function and start to contribute to disease progression. She will also discuss how understanding more about these cells and inflammation could be key in the development of new potential therapeutic strategies for ALS/MND.
Whilst Dr Krix’s talk will look at the understanding the biology behind neuroinflammation, Dr Stanley Appel’s talk will focus on a potential therapy for the treatment of ALS/MND. Dr Appel is the Direction of the Johnson Centre for Cellular Therapeutics at Houston Methodist Research Institute.
Regulatory T cells (Tregs) play a crucial role in regulating the body’s immune responses by restoring the activity of the immune system to normal and helping to prevent unnecessary inflammation. There is growing evidence that the number of Tregs are dramatically decreased in those with ALS/MND and they are less effective in suppressing the immune response. It has been proposed that the use of low-dose Interleukin-2 (IL-2), which is vital for the survival and function of Tregs, could increase the number of protective Tregs, reduce neuroinflammation and help to treat ALS. In Dr Appel’s talk ‘Regulatory T Lymphocyte Immunomodulatory Therapy for ALS’ (C22) he will discuss the use and results of IL-2 in Phase 1 and Phase 2A clinical trials for the treatment of ALS.
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Day 4, Session 9: Trial Design and Clinical Endpoints
There have been hundreds of clinical trials for ALS/MND all over the world, but at present there is only one drug approved for MND in the UK and three approved in the US. This has led to increased discussions over the design of clinical trials, such as trial length and the endpoints used to show whether the new drug being tested has any benefit for people living with ALS/MND. This session will delve deeper into these discussions and give perspectives from a range of different researchers.
Dr Ruben van Eijk, from University Medical Center Utrecht, will kick of the session with a talk titled ‘Improving clinical endpoints in therapeutic trials in ALS’ (C24). As ALS/MND is a complex heterogenous disease, meaning that it affects individuals very differently, trying to find sensitive measures of disease progression (endpoints) which can help to determine the effectiveness of new potential treatments is difficult. In his talk Dr van Eijk, will discuss the current endpoints available for ALS/MND clinical trials, their limitations and considerations on how trials could be improved for future studies.
Next up, are Dr Toby Ferguson and Stephanie Fradette from Biogen with their talk ‘Improving ALS Clinical Trial Design’ (C25). In their presentation, Dr Ferguson and Ms Fradette will discuss the challenges of clinical trial design and opportunities to improve and optimise trial design. They will also discuss how biomarkers, which are biological signals of the disease, have an important role to play in trial design in the future. Their talk will focus on the experience and learnings that could be taken from the clinical trials for Tofersen, a potential drug for the treatment of SOD1 MND, and BIIB078, which was designed for the treatment of C9orf72 MND.
Day 4, Session 10: Closing Session
The final talk of this year’s Symposium is from Dr Leigh Hochberg, from Massacheusetts general Hospital, Brown University, Harvard Medical School and US Department of Veterans Affairs. Dr Hochberg is the Principal Investigator of the BrainGate clinical trial, which is investigating a new technology which uses intracortically-based Brain-Computer Interfaces (iBCIs). This device has been developed to allow people who are paralysed to use their brain to control communication devices and assistive technologies. In his talk titled ‘BrainGate: Clinical Trials in Intracortical Brain-Compute Interfaces Toward the Restoration of Communication and Mobility’ (C27) Dr Hochberg will review the recent progress in iBCIs, as well as the challenges and opportunities of using this type of technology.
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