University of Miami Researchers Develop Biomarkers for Early ALS Detection
Researchers at the University of Miami Miller School of Medicine, led by Michael Benatar, M.D., Ph.D., and Joanne Wuu, Sc.M., have made significant progress in the early detection of amyotrophic lateral sclerosis (ALS) through the development of blood-based biomarkers. The Pre-Symptomatic Familial ALS (Pre-fALS) study, initiated in 2007, has been pivotal in collecting longitudinal data and biological samples from individuals at genetic risk for ALS. The discovery of neurofilament light chain (NfL) as a biomarker has been crucial in predicting the onset of ALS symptoms, allowing for earlier intervention. This advancement is expected to enhance therapeutic development efforts, which have previously faced challenges due to delayed intervention. The study has also led to the ATLAS trial, in collaboration with Biogen, focusing on treating individuals with a pathogenic variant in the SOD1 gene using an antisense oligonucleotide before clinical signs of ALS appear.