Debamestrocel (NurOwn / MSC-NTF) for amyotrophic lateral sclerosis
phase 3drug program · high · Mon Jun 30 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate safety and efficacy of autologous MSC-NTF cell therapy for ALS, including early symptomatic ALS with moderate disease presentation.
Autologous bone-marrow-derived mesenchymal stromal cells induced to secrete neurotrophic factors, administered intrathecally; randomized placebo-controlled trials with optional open-label extension in the ENDURANCE study.
ENDURANCE phase 3 trial registered to compare Debamestrocel - MSC-NTF (NurOwn) versus placebo, followed by open-label NurOwn dosing for all participants.
Prior randomized phase 3 ALS study did not meet the primary endpoint; MSC-NTF was well tolerated, with biomarker changes and a prespecified less-severe subgroup showing directional clinical response signals. A 2024 biomarker analysis reported debamestrocel-driven changes across neuroinflammation, neurodegeneration, and neuroprotection pathways, including reduced NfL associated with less ALSFRS-R decline.
Debamestrocel ALS CSF biomarker response program
exploratorybiomarker · medium · Mon Jan 01 2024 00:00:00 GMT+0000 (Coordinated Universal Time)
Assess whether CSF biomarkers can characterize debamestrocel biological activity and predict clinical outcomes in ALS.
Analysis of 45 CSF biomarkers from ALS trial participants, including subgroup analyses, prespecified outcome-prediction modeling, and causal inference relating NfL to ALSFRS-R decline.
2024 Muscle & Nerve publication reported exploratory CSF biomarker findings from 196 participants.
Debamestrocel changed 64% of biomarkers studied, spanning neuroinflammation, neurodegeneration, and neuroprotection pathways. Baseline NfL, baseline LAP/TGF-beta1, and change in galectin-1 were predictive of clinical outcomes in debamestrocel-treated participants; NfL reduction was associated with less ALSFRS-R decline.
NurOwn / MSC-NTF cells for progressive multiple sclerosis
phase 2drug program · high · Sun Jan 01 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate safety and efficacy of repeated intrathecal NurOwn treatment in participants with progressive multiple sclerosis.
Open-label multidose study of autologous mesenchymal stromal stem cells secreting neurotrophic factors, with three intrathecal cell treatments and clinical, MRI, safety, and CSF biomarker assessments.
Open-label phase 2 progressive MS study completed with published results; authors stated further confirmation in a randomized study is warranted.
No deaths, MS worsening adverse events, MRI disease activation, or clinically significant safety lab changes were reported. Two participants developed symptoms consistent with arachnoiditis. Nineteen percent achieved prespecified walking or hand-function improvements, with CSF neuroprotective factors increased and inflammatory biomarkers decreased.
NurOwn MSC-NTF cell therapy platform
undisclosedplatform · high
Produce autologous MSC-NTF cells intended for neurodegenerative disease therapy.
Technology platform for generating commercial-ready autologous mesenchymal stromal cells induced to secrete neurotrophic factors.
Company lists NurOwn as its pipeline platform and states the autologous MSC-NTF therapy remains investigational and is not FDA approved.
