<div class="infobox infobox-company">
<div class="infobox-header">BrainStorm Cell Therapeutics Inc.</div>
<div class="infobox-row"><strong>Headquarters:</strong> New York, New York, USA</div>
<div class="infobox-row"><strong>Founded:</strong> 2000</div>
<div class="infobox-row"><strong>Public:</strong> NASDAQ (BCLI)</div>
<div class="infobox-row"><strong>Market Cap:</strong> ~$50M USD</div>
</div>
Overview
flowchart TD
therapeutics["therapeutics"] -->|"protects against"| age_related_cognitive_decline["age-related cognitive decline"]
therapeutics["therapeutics"] -->|"inhibits"| neuroinflammation["neuroinflammation"]
Therapeutics["Therapeutics"] -->|"references"| SIRT6["SIRT6"]
Therapeutics["Therapeutics"] -->|"references"| AADC["AADC"]
Therapeutics["Therapeutics"] -->|"references"| CX3CR1["CX3CR1"]
Therapeutics["Therapeutics"] -->|"references"| BACE1["BACE1"]
Therapeutics["Therapeutics"] -->|"references"| APOE["APOE"]
Therapeutics["Therapeutics"] -->|"references"| VCP["VCP"]
Therapeutics["Therapeutics"] -->|"references"| GFAP["GFAP"]
Therapeutics["Therapeutics"] -->|"references"| NURR1["NURR1"]
Therapeutics["Therapeutics"] -->|"references"| BDNF["BDNF"]
Therapeutics["Therapeutics"] -->|"references"| NLRP3["NLRP3"]
Therapeutics["Therapeutics"] -->|"references"| TFEB["TFEB"]
Therapeutics["Therapeutics"] -->|"references"| PPARGC1A["PPARGC1A"]
style therapeutics fill:#4fc3f7,stroke:#333,color:#000
...<div class="infobox infobox-company">
<div class="infobox-header">BrainStorm Cell Therapeutics Inc.</div>
<div class="infobox-row"><strong>Headquarters:</strong> New York, New York, USA</div>
<div class="infobox-row"><strong>Founded:</strong> 2000</div>
<div class="infobox-row"><strong>Public:</strong> NASDAQ (BCLI)</div>
<div class="infobox-row"><strong>Market Cap:</strong> ~$50M USD</div>
</div>
Overview
Mermaid diagram (expand to render)
BrainStorm Cell Therapeutics Inc. is a biotechnology company pioneering the development of autologous cell therapies for neurodegenerative diseases. The company's lead product, NurOwn®, is a personalized cell therapy derived from the patient's own bone marrow stromal cells (MSCs) that are engineered to secrete high levels of neurotrophic factors. BrainStorm's approach represents a novel therapeutic strategy that combines cell therapy with gene therapy concepts, using the patient's own cells as a manufacturing platform for delivering regenerative factors directly to the central nervous system["@brainstorm"].
The company focuses on diseases where neurotrophic factor deficiency plays a key role in disease progression, particularly amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Alzheimer's disease. BrainStorm's technology platform addresses a critical unmet need in neurodegenerative disease — the progressive loss of neuronal function due to reduced trophic support.
MSC-NTF Cell Therapy
BrainStorm's proprietary technology platform, MSC-NTF (Mesenchymal Stromal Cells engineered to secrete NeuroTrophic Factors), represents a significant advancement in cell-based therapeutics:
- Cell Source: Autologous bone marrow-derived mesenchymal stromal cells from the patient
- Engineering Process: Cells are cultured under proprietary conditions that induce them to secrete elevated levels of neurotrophic factors including:
- Brain-Derived Neurotrophic Factor (BDNF)
- Glial Cell Line-Derived Neurotrophic Factor (GDNF)
- Vascular Endothelial Growth Factor (VEGF)
- Hepatocyte Growth Factor (HGF)
- Delivery Method: Intrathecal injection into the cerebrospinal fluid, allowing direct delivery to the spinal cord and brain
Mechanism of Action
The therapeutic benefit of NurOwn derives from multiple mechanisms:
Neurotrophic Support: Direct delivery of BDNF and GDNF supports motor neuron survival and function
Immunomodulation: MSCs exert anti-inflammatory effects on the local immune environment
Neuroprotection: Combined factors protect [neurons](/entities/neurons) from excitotoxicity and oxidative stress
Potential for Regeneration: Some evidence suggests support for neural repair processesPipeline / Products
| Program | Target/Mechanism | Indication | Phase | Status |
|---------|-----------------|------------|-------|--------|
| NurOwn (MSC-NTF) | Autologous MSC-NTF cells | ALS | Phase 3 | Completed |
| NurOwn | Autologous MSC-NTF cells | Progressive MS | Phase 1 | Active |
| NurOwn | Autologous MSC-NTF cells | [Alzheimer's Disease](/diseases/alzheimers-disease) | Preclinical | Planning |
Clinical Development
ALS (Amyotrophic Lateral Sclerosis)
NurOwn has undergone extensive clinical evaluation in ALS:
Phase 2 Trial (2018): An open-label, single-arm study demonstrated safety and suggested efficacy. Patients showed slower progression on the ALSFRS-R scale compared to historical controls, with responders identified as those maintaining or improving function[@nurown].
Phase 3 Trial (2020-2022): The pivotal Phase 3 trial enrolled approximately 200 patients with ALS at multiple U.S. sites. While the trial did not meet its primary endpoint of a statistically significant difference in response rate at 28 weeks, pre-specified subgroup analyses revealed clinically meaningful benefits in patients with less advanced disease (baseline ALSFRS-R ≥ 35). These patients showed significantly better outcomes in function, breathing, and quality of measures[@nurowna].
Multiple Sclerosis
BrainStorm is conducting an open-label Phase 1 trial evaluating NurOwn in patients with progressive forms of MS. The trial aims to assess safety and explore potential neuroprotective effects in a disease characterized by demyelination and neuronal loss.
[Alzheimer's Disease](/diseases/alzheimers-disease)
Preclinical studies have explored NurOwn in Alzheimer's disease models, demonstrating reduced neuroinflammation and improved cognitive function. The company has announced plans to advance this program based on these findings.
Funding & Financials
BrainStorm has pursued a strategic funding approach to support its clinical programs:
- 2000: Company founded
- 2014: Initial public offering on NASDAQ
- 2021: $15M public offering to support Phase 3 trial completion
- 2022: $30M registered direct offering for ongoing operations and pipeline development
- Current Market Cap: Approximately $50M USD (as of 2026)
Key People
- Dr. Ralph L. Kern (President & Chief Executive Officer) — Brings extensive experience in neuroscience drug development
- Dr. William J. R. Schmidt (Chief Medical Officer) — Oversees clinical development and regulatory strategy
Competitive Landscape
BrainStorm's cell therapy approach occupies a unique position in the ALS treatment landscape:
- Traditional Small Molecules: Riluzole and edaravone remain standard of care
- Novel Mechanisms: ASO therapies (e.g., tofersen for SOD1), antisense approaches
- Other Cell Therapies: Various companies exploring MSC-based approaches, though BrainStorm's MSC-NTF platform is most advanced
- Gene Therapy: AAV-based approaches targeting specific genetic mutations
The autologous nature of NurOwn provides potential advantages in safety and immune tolerance compared to allogeneic or synthetic therapies.
Challenges and Future Directions
BrainStorm faces several challenges and opportunities:
Phase 3 Interpretation: The mixed results require careful analysis of patient subgroups and potential paths toward regulatory approval
Manufacturing Scale: Autologous cell therapies present manufacturing and logistical challenges
Regulatory Pathway: Working with FDA on potential accelerated approval based on subgroup findings
Pipeline Expansion: MS and Alzheimer's programs offer additional value driversSee Also
- [Amyotrophic Lateral Sclerosis](/diseases/amyotrophic-lateral-sclerosis) (ALS)
- [Cell Therapy for Neurodegeneration](/therapeutics/cell-therapy-neurodegeneration)
- [Neurotrophic Factors](/mechanisms/neurotrophic-factors)
- [Mesenchymal Stem Cells](/therapeutics/mesenchymal-stem-cell-therapy)
- [ALS Treatment Pipeline](/companies/als-pipeline-companies)
References
Unknown, BrainStorm Cell Therapeutics Corporate Website (n.d.)
[Unknown, NurOwn Phase 2 ALS Trial Results (n.d.)](https://pubmed.ncbi.nlm.nih.gov/29368666/)
[Unknown, NurOwn Phase 3 ALS Trial Results (n.d.)](https://pubmed.ncbi.nlm.nih.gov/36789012/)