May 4, 2021
The key to optimal treatment of amyotrophic lateral sclerosis (ALS) may be to initiate treatment
before symptoms emerge a difficult, but intriguing, hypothesis that will soon be tested in a clinical trial.
That trial, known as ATLAS, will evaluate the best timing to begin treatment with the investigational antisense oligonucleotide tofersen in presymptomatic
SOD1 mutation carriers at high risk for ALS.
The ATLAS trial will assess levels of the axonal injury marker neurofilament light (NfL) to identify imminent phenoconversion in superoxide dismutase 1 (
SOD1) mutation carriers, then randomize participants to treatment or placebo arms, reported Michael Benatar, MD, PhD, of the University of Miami in Florida, and co-authors in a presentation at the 2021 American Academy of Neurology annual meeting.