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Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases
Brain  (IF13.501),  Pub Date : 2021-10-22, DOI: 10.1093/brain/awab404
Michael Benatar, Joanne Wuu, Caroline McHutchison, Ronald B Postuma, Bradley F Boeve, Ronald Petersen, Christopher A Ross, Howard Rosen, Jalayne J Arias, Stephanie Fradette, Michael P McDermott, Jeremy Shefner, Christine Stanislaw, Sharon Abrahams, Stephanie Cosentino, Peter M Andersen, Richard S Finkel, Volkan Granit, Anne-Laure Grignon, Jonathan D Rohrer, Corey T McMillan, Murray Grossman, Ammar Al-Chalabi, Martin R Turner, the attendees of the First International Pre-Symptomatic ALS Workshop

Significant progress has been made in understanding the pre-symptomatic phase of amyotrophic lateral sclerosis (ALS). While much is still unknown, advances in other neurodegenerative diseases offer valuable insights. Indeed, it is increasingly clear that the well-recognized clinical syndromes of Alzheimer’s disease (AD), Parkinson’s disease (PD), Huntington’s disease (HD), spinal muscular atrophy, and frontotemporal dementia are also each preceded by a pre-symptomatic or prodromal period of varying duration, during which the underlying disease process unfolds, with associated compensatory changes and loss of inherent system redundancy. Key insights from these diseases highlight opportunities for discovery in ALS. The development of biomarkers reflecting amyloid and tau has led to a shift in defining AD based on inferred underlying histopathology. PD is unique among neurodegenerative diseases in the number and diversity of non-genetic biomarkers of pre-symptomatic disease, most notably REM-sleep behavior disorder. HD benefits from an ability to predict the likely timing of clinically manifest disease based on age and CAG-repeat length alongside reliable neuroimaging markers of atrophy. SMA clinical trials have highlighted the transformational value of early therapeutic intervention, and studies in FTD illustrate the differential role of biomarkers based on genotype. Similar advances in ALS would transform our understanding of key events in pathogenesis, thereby dramatically accelerating progress towards disease prevention.