Filaments line up as MS biomarker

Why neurofilaments could become the go-to biomarker for neurodegeneration

Neurofilament assays appear on track to become the biomarker of choice for a wide range of neurodegeneration studies, given their tight correlation with axon damage and detectability in blood.

If prospective trials can reinforce the positive data from retrospective clinical studies, the biomarker could see wide uptake in multiple sclerosis and beyond.

As researchers search for biomarkers to supersede the symptom-based diagnostics and clinical trial endpoints that limit progress in neurology, blood-based readouts rise to the top of the wish list because they are less invasive than obtaining CSF via spinal taps, and cheaper than imaging.

Academic and industry researchers are ramping up activity on neurofilaments as a biomarker, because their linkage with axon degeneration would find use in multiple sclerosis, Alzheimer’s disease and a host of other neurodegenerative indications.

“This is the first biomarker showing robust correlation with neuronal injury in blood and it works across diseases,” Henrik Zetterberg, an academic expert on AD biomarkers, told BioCentury. Zetterberg is a professor and chief physician at the University of Gothenburg and professor of neurochemistry at University College London.

In February, FDA released guidance stating it is open to a biomarker-based endpoint for trials of early stage AD when symptoms aren’t yet present, although it did not name a specific biomarker.

In April, NIH’s National Institute on Aging (NIA) and the Alzheimer’s Association released a framework advocating for a biological definition of the disease and recommending a composite biomarker dubbed the “ATN system” that involves

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