Neurofilament biomarker in amyotrophic lateral sclerosis


  • 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.