Serum neurofilament light (NfL) and serum phosphorylated neurofilament heavy (pNfH) likely act as prognostic and phamacodynamic biomarkers in amyotrophic lateral sclerosis (ALS).
Why this matters ?
Phase II studies for ALS therapies usually use survival or rate of decline as the primary outcome; however, most studies are typically underpowered for such testing, making it difficult to decide which therapies to take to phase III.
Controlling for prognostic and pharmacodynamic biomarkers may help overcome these challenges.
NfL and pNfH have previously been proposed as diagnostic, prognostic and pharmacodynamic biomarkers, however, questions remain around their use.