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Also known as MSC-NTF cells transplantation (IM), MSC-NTF cells transplantation, NurOwn (MSC-NTF cells), Debamestrocel - MSC-NTF (NurOwn), Debamestrocel- MSC-NTF
NurOwn · 6 trials · 3 indications
To evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS based on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) which is a 12-item clinician-administered questionnaire. Each item is scored between 0 and 4, with the total ALSFRS-R score between 0 and 48 where a higher score reflects a better outcome.
To evaluate the safety and tolerability of NurOwn in the treatment of participants with ALS throughout the study including the DBPC period (Part A) to Week 24 and the Open Label Extension (Part B).
The ALSFRS-R is a quickly administered (10 minutes) ordinal, validated rating scale (ratings 0-4) used to determine participants' assessment of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, as measured by quantitative neuromuscular strength testing, and with quality of life measures, and predicted survival. The total score of ALSFRS-R ranges from 0-48, with higher score being better.
Combined safety of all 3 intrathecal doses of NurOwn® (MSC-NTF cells) Number of participants who experienced Treatment-emergent Adverse Events during the study. Treatment-emergent Adverse Event is an adverse event that occurs for the first time after initiation of first treatment or if it had occurred prior to initiation first treatment, it worsens in severity after initiation of first treatment.
Safety assessed based on the incidence of treatment-emergent adverse events (TEAEs) (including serious adverse events \[SAEs\]) including clinically relevant changes in vital signs, clinical laboratory assessments, physical and neurological examinations, and electrocardiogram (ECG) tests, during transplantation of expanded autologous MSC-NTF cells administered on a single occasion via combined intrathecal (IT) administration and intramuscular (IM) injections
Safety evaluation and tolerability of a single treatment administration of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF) by multiple intramuscular injections (IM) at 24 separate sites on the biceps and triceps muscles to patients with ALS at the early disease stage
Safety evaluation and tolerability of single intrathecal injection (IT) into the cerebrospinal fluid (CSF) of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF),to patients with ALS at the progressive disease stage.
| Arm | Type | Description |
|---|---|---|
| Debamestrocel - MSC-NTF (NurOwn) | EXPERIMENTAL | NTF-secreting mesenchymal stem cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients |
| Placebo | PLACEBO_COMPARATOR | Placebo is comprised of Dulbecco Modified Eagle Medium (DMEM) |
| NurOwn® (MSC-NTF cells) | ACTIVE_COMPARATOR | Three Intrathecal administrations of NurOwn® (MSC-NTF cells) at bi-monthly intervals |
| NurOwn (MSC-NTF cells) | EXPERIMENTAL | Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors |
| Nurown MSC-NTF cells | ACTIVE_COMPARATOR | Single autologous MSC-NTF cells treatment by combined intramuscular and intrathecal administration |
| Excipient | PLACEBO_COMPARATOR | Combined intramuscular and intrathecal placebo administration |
| MSC-NTF | EXPERIMENTAL | - |
| MSC-NTF cells IM | EXPERIMENTAL | Intramuscular administration in early stage patients |
| MSC-NTF cells IT | EXPERIMENTAL | Intrathecal administration in progressive stage patients |
| Name | Type | Description |
|---|---|---|
| Debamestrocel - MSC-NTF (NurOwn) | BIOLOGICAL | MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM). |
| Placebo | BIOLOGICAL | Dulbecco Modified Eagle Medium (DMEM). |
| NurOwn® (MSC-NTF cells) | BIOLOGICAL | NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10\^6 cells every 8 weeks NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors |
| Bone Marrow aspiration | OTHER | Bone Marrow aspiration |
| NurOwn (MSC-NTF cells) | BIOLOGICAL | Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors |
| Nurown MSC-NTF cells | BIOLOGICAL | Single autologous MSC-NTF cells treatment by combined intramuscular and intrathecal administration |
| MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF | BIOLOGICAL | - |
| MSC-NTF cells transplantation (IM) | BIOLOGICAL | In early ALS subjects: Autologous MSC-NTF cells will be transplanted under mild anesthesia, intramuscularly into patients' clinically unaffected (or only mildly affected) upper arm biceps and triceps muscles according to a pre-designed grid. Intramuscular injections will be by a 26 gauge needle to a 1.5cm depth (ensuring that injection is into muscle and not adipose tissue). The patients will be injected at 24 sites with a total of 24 million cells |
| MSC-NTF cells transplantation (IT) | BIOLOGICAL | In progressive ALS subjects: Autologous MSC-NTF cells will be transplanted under mild anesthesia intrathecally(via a standard lumbar puncture)with a total of 60 million cells. |
Inclusion Criteria: 1. Male and female participants 18 to 75 years old (inclusive) at Screening Visit 1. 2. ALS diagnosed as laboratory-supported probable, clinically probable, or definite as defined by the revised El Escorial criteria. 3. Having onset of ALS symptoms, including muscle weakness, wi...
NurOwn is being developed for amyotrophic lateral sclerosis (ALS) and chronic progressive multiple sclerosis. Brainstorm Cell Therapeutics is studying it in ALS and in progressive MS, both neurological conditions. It is an investigational therapy and has not been approved for commercial use.
NurOwn is developed by Brainstorm Cell Therapeutics Inc., which trades under the ticker BCLI. The company holds Orphan Drug and Fast Track designations from the FDA for the program. Development spans ALS and chronic progressive multiple sclerosis.
NurOwn is in Phase 3 development. A completed Phase 3 trial in ALS enrolled 196 participants, and a Phase 3 ALS study called ENDURANCE is planned with 200 participants but is not yet recruiting. The drug remains investigational and has not been approved by the FDA.
NurOwn has been studied across five trials. Key studies include NCT03280056, a completed Phase 3 ALS trial with 196 participants; NCT02017912, a completed Phase 2 ALS trial with 48 participants; NCT03799718, a completed Phase 2 trial in progressive MS with 23 participants; and NCT06973629, the Phase 3 ENDURANCE ALS study, which is not yet recruiting.
Yes. NurOwn is also known as debamestrocel, or MSC-NTF cells. Other names used for the same asset include MSC-NTF cells transplantation, NurOwn (MSC-NTF cells), and Debamestrocel-MSC-NTF. These terms refer to the same investigational cell therapy developed by Brainstorm Cell Therapeutics.
Across its clinical program, NurOwn trials have enrolled a total of 470 participants. The largest completed study, NCT03280056, randomized 196 ALS patients in a Phase 3 trial. The planned Phase 3 ENDURANCE study, NCT06973629, is designed to enroll 200 participants with early symptomatic ALS.