Wellcome Trust Senior Research Fellow, Honorary Consultant Neurologist Department of Basic and Clinical Neuroscience, IoPPN, King’s College London, UK

Progress in understanding and treating ALS-FTD

Host: E. Buratti

Amyotrophic lateral sclerosis (ALS) is the most common and aggressive adult-onset motor neuron disease. Management relies on multidisciplinary clinical care and is largely palliative. ALS remains incurable with most patients dying within 3-5 years of symptom onset. Importantly, ALS overlaps with frontotemporal dementia (FTD), a form of dementia that is particularly common in younger people. Characterisation of gene mutations linked to ALS-FTD have led to important advances in our understanding of motor neuron degeneration and dementia. Though invariably fatal conditions, recent clinical trials in ALS have resulted in the exciting development of a new gene therapy for SOD1 linked ALS. However, much work still needs to be done to understand and treat the remaining ~97% of ALS, which is characterised by misregulation of the DNA/RNA binding protein, TDP-43. In this presentation I will summarise the history of ALS research, our current understanding of TDP-43 linked ALS-FTD and the unmet needs that remain in our field.


Hundreds of videos, tens of playlists providing free scientific content worldwide