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NIH Record - 75th Anniversary - National Institutes of Health

Scientists Discover Urinary Biomarker That May Help Track ALS

A study in Neurology suggests that analyzing levels of the protein p75ECD in urine samples from people with amyotrophic lateral sclerosis (ALS) may help monitor disease progression as well as determine the effectiveness of therapies. The study was supported by NINDS and NCATS.

Dr. Mary-Louise Rogers, senior research fellow at Flinders University in Adelaide, Australia, and Dr. Michael Benatar, professor of neurology at the University of Miami, and their teams discovered that levels of urinary p75ECD increased gradually in patients with ALS as their disease progressed over a 2-year study period.

“It was encouraging to see changes in p75ECD over the course of the study, because it suggests an objective new method for tracking the progression of this aggressive disease,” said Dr. Amelie Gubitz, program director at NINDS. “In addition, it indicates the possibility of assessing whether levels of that protein decrease while patients try future treatments, to tell us whether the therapies are having any beneficial effects.”

Further analysis of the samples from 54 patients revealed that those who began the study with lower levels of urinary p75ECD survived longer than did patients who had higher levels of the protein initially, suggesting that it could be a prognostic marker of the disease and may inform patients about their illness. Benatar and his team noted that this may be useful in selecting participants for clinical trials and in improving study design.     

ALS is a fatal neurodegenerative disease in which motor neurons—cells that control muscle activity such as walking, talking and breathing—gradually die off, resulting in paralysis. There is no cure for ALS.

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