Publications by authors named "Y Gothelf"
Article Synopsis
- This study looked at how a treatment called debamestrocel affects special substances in the body (biomarkers) in people with ALS, a disease that affects muscles.
- They tested 196 people, some got the treatment while others got a fake treatment (placebo) and checked their cerebrospinal fluid (CSF) for 45 different biomarkers.
- Results showed that debamestrocel improved many of these biomarkers, which could mean it's helping to protect nerves and reduce inflammation, especially in patients with more advanced ALS.
Background: Autologous mesenchymal stem cell neurotrophic factor-secreting cells (NurOwn) have the potential to modify underlying disease mechanisms in progressive multiple sclerosis (PMS).
Objective: This open-label phase II study was conducted to evaluate safety/efficacy of three intrathecal cell treatments.
Methods: Eighteen participants with non-relapsing PMS were treated.
Introduction/aims: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative illness with great unmet patient need. We aimed to evaluate whether mesenchymal stem cells induced to secrete high levels of neurotrophic factors (MSC-NTF), a novel autologous cell-therapy capable of targeting multiple pathways, could safely slow ALS disease progression.
Methods: This randomized, double-blind, placebo-controlled study enrolled ALS participants meeting revised El Escorial criteria, revised ALS Functional Rating Scale (ALSFRS-R) ≥25 (screening) and ≥3 ALSFRS-R points decline prior to randomization.
Effects of MSC-NTF cells on T and B regulatory cell function in ALS.
Amyotroph Lateral Scler Frontotemporal Degener
November 2020
Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disorder with complex biology and significant clinical heterogeneity. Many preclinical and early phase ALS clinical trials have yielded promising results that could not be replicated in larger phase 3 confirmatory trials. One reason for the lack of reproducibility may be ALS biological and clinical heterogeneity.
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