Blood protein tags progression in ALS

In a study sponsored by the ALS Association, researchers have assessed a blood protein biomarker that could be used to track the progression of amyotrophic lateral sclerosis (ALS). ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disease leading to total paralysis, with death often resulting within two to 5 years of diagnosis. The only FDA-approved treatment for ALS is Sanofi-Aventis' ($SNY) Rilutek (riluzole), and this extends survival by just a few months on average.

The protein, pNF-H (phosphorylated neurofilament heavy subunit), is one of the components of nerves. It is also found in the cerebrospinal fluid (CSF), but testing this is invasive and uncomfortable, and is associated with headaches and nausea. Because of this, a blood biomarker would be a safer and more convenient alternative.

The researchers measured the concentration of the protein in blood and CSF over a year, and found that higher levels were linked with faster decline and disease progression, and a greater risk of death during the 12 months of the study. The results were published online in the Journal of Neurology, Neurosurgery & Psychiatry.

If these results can be validated, doctors could use the biomarker as a more objective way to track the progression of disease and see whether patients are responding to therapy than the currently used ratings scales. It could also play a role in drug development and clinical trials.

"Although a biomarker is only proven once tested in a clinical trial and shown to be more predictive than the currently available measure of progression in ALS, this protein appears to be a very promising biomarker of disease progression in a relatively large group of patients and should allow us to conduct shorter clinical trials and speed the search for new therapies," says Lucie Bruijn, chief scientist for the ALS Association.

The study is part of the ALS Association's Translational Research Advancing Therapies (TREAT ALS) research portfolio.

- read the press release
- see the abstract

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