The global MND research community is once again preparing for one of the most anticipated events of the year—the 36th International Symposium on ALS/MND. This annual gathering is the largest scientific and medical conference dedicated solely to ALS/MND, offering an international platform to share work, exchange ideas, and spark new collaborations. Throughout November, we’ll be publishing a series of blog posts to highlight some of the exciting research being presented at this year’s Symposium.
Every year, the International Symposium on ALS/MND features a lineup of plenary speakers who are leading experts in their fields. This year, we’re excited to welcome 23 plenary speakers, each bringing their unique expertise to the global stage. Over the coming weeks, we’ll be shining a spotlight on each of these speakers and offering a glimpse into what they’ll be discussing at the Symposium.
This year, the Symposium will be held in San Diego, USA, from 5–7 December, with a virtual attendance option available. Virtual delegates will be able to join select sessions live and access all sessions on demand after the event—just like in-person attendees. We’re proud to once again bring this important event to the community and look forward to welcoming researchers, healthcare professionals and people from the wider MND community. Haven’t registered yet? There’s still time!
Day 2: Session 6a Proteostasis & Proteotoxicity
A potential new target for MND treatments
TDP-43 is a protein which is affected in MND. In the majority of cases of MND, TDP-43 protein forms abnormal clumps in the cells. In healthy cells, TDP-43 is found in the nucleus, the command centre of the cell which contains our DNA. In MND, it is found in the wrong part of the cell, which means it cannot carry out the job that it is supposed to do. This also happens in other age-related neurodegenerative diseases.
Dr Don Cleveland will talk about his work into TDP-43 and how this could be used to develop new treatments for MND. In MND, TDP-43 may be prevented from getting into the nucleus by the addition of a small molecule to the protein. This addition could stop TDP-43 from being imported into the nucleus, leading to a build-up of TDP-43 in other parts of the cell. Dr Cleveland and his team found that a protein, called CREBBP acetyltransferase, may cause the addition of this extra molecule to TDP-43. CREBBP acetyltransferase was also found to be increased in MND neurons.
A potential strategy for treating MND is to find ways to get TDP-43 into the nucleus. Dr Cleveland will discuss how this could be achieved by changing the TDP-43 so it can enter the nucleus in other ways. This would mean that there would be more TDP-43 in the nucleus, where it can carry out its job effectively. Dr Cleveland will highlight how this could be a promising strategy for treating MND.
Day 1: Session 2a Modelling And Treating ALS With Stem Cells
Treating MND with stem cells
Stem cells have the potential to become multiple different types of cell. There has been much excitement about the possibility of using stem cells as a treatment for MND. This could mean cell replacement therapy, where new cells are introduced to the brain and/or spinal cord that may be able to replace the cells which have died in MND. Some types of stem cells also release factors which promote repair and survival of other cells, and some therapies involve making these stem cells release factors which could support and improve the growth and survival of motor neurons.
An expert panel will discuss the uses and potential of stem cells in MND research and as a potential treatment for MND. Prof Clive Svendsen and Prof Merit Cudkowicz will start the panel with an introduction to stem cells including the opportunities and challenges they present. Dr Jonathan Glass will then discuss lessons which have been learned from clinical trials using stem cells. Past and current trials will be explored, including both cell replacement approaches, and stem cells which have been changed to release powerful factors to support motor neuron health and survival. Exploring what has been successful and what could be improved in past trials will help to inform future trials of stem cell treatments in MND.
Day 3: Session 9b Therapeutic Strategies
Using machine learning to find new treatments for MND
Machine learning is a field of artificial intelligence (AI) focused on systems which can identify patterns and learn from data. Using machine learning, large amounts of data can be analysed much faster, and patterns can be found which may be missed by people. Collecting and analysing data on this scale could speed up the process of discovering new treatments for MND.
Dr Samuel Sances from Insitro will be discussing how they are using machine learning and some of things they have learnt from this approach to drug discovery in MND. They generate large volumes of data from MND stem cells using a range of different experiments. The machine learning technology will then identify patterns in the data to find common changes which occur in MND. This approach has uncovered ALS-1, a potential target for MND treatment which could improve motor neuron health. Dr Sances will be discussing this discovery and how machine learning can reveal new biological insights and identify new targets and treatments for MND.
Day 1: Session 3b Clinical Trials And Trial Design
Advances in SMA treatment: Lessons for MND
An important part of MND research is learning from research into other conditions to see what can also be applied to MND. Dr Charlotte Sumner from John Hopkins Medicine will discuss her work in spinal muscular atrophy (SMA), and what lessons can be learned for MND from this field.
SMA is a rare genetic condition that causes muscle weakness. Symptoms usually appear in babies and toddlers. SMA is caused by a change in the SMN gene, which is important for motor neuron survival. There are currently three treatments available which target the SMN gene and can improve the symptoms of SMA. All of these treatments work better when given to newborn babies with changes in the SMN gene rather than waiting for symptoms to appear. However, children with SMA can still have significant symptoms, likely because the disease process begins before the child is born.
While the development of treatments for SMA is a remarkable success, it has also raised multiple new questions. Dr Sumner will be discussing ongoing studies into understanding how SMA develops, efforts to improve the current treatments, and whether treatments can be given prenatally. Many of the questions raised by SMA treatments also have relevance to MND, as there are several similarities between the two conditions. Insights from SMA may inspire new avenues for MND research.
Day 3: Session 10 Joint Closing Session
Finding treatments for everyone with MND
We know that MND doesn’t affect everyone in the same way. People can have different symptoms, the disease can progress at different speeds, and survival times can vary. Doctors and researchers understand this, but clinical trials often treat MND as if it’s one single condition. This can be a problem as a treatment might seem like it doesn’t work in a trial, even though it could help some people with MND.
In the closing session of this year’s symposium, Dr Jeffrey Rosenfeld from Loma Linda University Health will discuss why it’s important to recognise these differences when testing new treatments in clinical trials. He and his team have looked at large sets of data from people with MND and found that there are different subgroups of the disease, based on how fast it progresses and how long people live with it. Dr Rosenfeld will explain how future clinical trials could use these subgroups to more accurately test whether treatments are beneficial and hopefully find effective treatments for everyone with MND.
Stay informed
You can find out more about the International Symposium on ALS/MND on the website and view the programme for this year’s event.
