BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, this week presented new biomarker data from the Phase 3 trial of its late-stage investigational ALS treatment, NurOwn at the 2023 ALS and Related Motor Neuron Diseases Gordon Research Conference. These data show that treatment with NurOwn significantly elevated markers of neuroprotection and lowered markers of neuroinflammation and neurodegeneration, including neurofilament light (NfL) over time compared to placebo in all trial participants.
New data analysis presented this week was motivated by the regulatory precedent set by a recent FDA drug approval in ALS, and the knowledge gained through the regulatory process, suggesting that the blood-based biomarker neurofilament light (NfL) is associated with disease prognosis and progression in patients with ALS and potentially other neurodegenerative diseases. It is believed that reductions in plasma NfL are reasonably likely to predict clinical benefit in ALS.
“It’s well known that in ALS, accounting for disease characteristics such as site of onset, time from first symptom to treatment and baseline physical function are important to understanding the treatment effect in clinical trials given the great heterogeneity in the disease, which can influence prognosis,” said Stacy Lindborg, PhD, BrainStorm co-CEO and presenter of the poster. “The data we presented at the Gordon Research Conference show that it is equally important to examine biomarker data, particularly neurofilament light, which is a predictor of disease progression. Treatment-driven reductions in NfL are associated with better clinical outcomes in ALS.”
SOURCE: PRNewswire