BrainStorm Therapeutics is a biotechnology company focused on developing novel therapies for amyotrophic lateral sclerosis (ALS) and Alzheimer's disease (AD). The company is headquartered in the United States and operates as a clinical-stage biotech dedicated to addressing unmet needs in neurodegenerative diseases.
BrainStorm's pipeline focuses on developing disease-modifying therapies for ALS and AD through innovative approaches targeting protein aggregation and neuroinflammation.
| Drug/Program |
Mechanism |
Target Indication |
Development Stage |
| NurOwn |
Cell therapy (MSC-NTF) |
ALS |
Phase 3 completed |
| NurOwn |
Cell therapy (MSC-NTF) |
AD |
Phase 2 |
| Next-gen platforms |
Protein aggregation modulators |
ALS/AD |
Discovery |
NurOwn is BrainStorm's proprietary cell therapy platform using mesenchymal stem cells (MSCs) engineered to secrete neurotrophic factors (NTFs). The technology involves:
- Autologous transplantation: Patient-derived stem cells
- Neurotrophic factor secretion: BDNF, GDNF, VEGF, HGF
- Anti-inflammatory properties: Modulation of microglia
- Neuroprotective effects: Promoting motor neuron survival
BrainStorm completed a Phase 3 randomized, double-blind, placebo-controlled trial of NurOwn in ALS patients. The trial evaluated the safety and efficacy of intrathecal delivery of NurOwn cells.
Key endpoints included:
- ALSFRS-R score progression
- Forced vital capacity (FVC)
- Survival outcomes
- Biomarker analysis (NFL levels)
The company is also developing NurOwn for Alzheimer's disease, leveraging the same mechanism of neurotrophic factor delivery to protect neurons and reduce amyloid/tau pathology.
- Founded: 2000s (clinical-stage company)
- Headquarters: United States
- Focus: ALS, Alzheimer's disease
- Technology: MSC-NTF cell therapy platform
- Status: Public company (NASDAQ: BCLI)
BrainStorm holds multiple patents covering:
- MSC-NTF cell manufacturing processes
- Delivery methods (intrathecal, intravenous)
- Combination therapies
- Biomarker signatures for patient selection
¶ Partnerships and Funding
The company has collaborated with academic institutions and has received funding through:
- NIH grants for ALS and AD research
- Strategic partnerships for clinical development
- Public market funding (NASDAQ)
¶ Competitive Landscape
BrainStorm competes with other cell therapy companies in ALS including:
BrainStorm's NurOwn technology leverages mesenchymal stem cells (MSCs) that have been engineered to over-express multiple neurotrophic factors. These factors include:
Beyond neurotrophic factor secretion, NurOwn cells exert immunomodulatory effects:
- Reduction in pro-inflammatory cytokines (IL-1β, TNF-α)
- Increase in anti-inflammatory cytokines (IL-10)
- Modulation of microglia toward M2 (protective) phenotype
- Reduction in reactive oxygen species (ROS)
¶ Amyloid and Tau Modulation
In Alzheimer's disease, NurOwn may provide:
- Reduced amyloid-beta plaque formation
- Decreased tau phosphorylation
- Protection against synaptic loss
- Improvement in neural connectivity
The Phase 3 trial enrolled 200 patients with ALS randomized 1:1 to receive NurOwn or placebo. Patients received 3 intrathecal administrations at 8-week intervals.
Primary Outcomes:
- Change in ALSFRS-R score at 28 weeks
- Change in forced vital capacity (FVC)
- Adverse event frequency
Key Findings:
- Clinically meaningful slowing of disease progression in pre-specified subgroups
- Favorable safety profile with no serious treatment-related adverse events
- Biomarker data showed reduction in neurofilament light chain (NFL) in treatment arm
NurOwn treatment was associated with:
- Decreased CSF NFL levels (marker of neuronal damage)
- Reduced inflammatory markers
- Increased anti-inflammatory cytokine levels
- Preserved gray matter volume on MRI
BrainStorm has developed a proprietary manufacturing process for NurOwn:
- Patient enrollment: Autologous cells from each patient
- MSC expansion: Large-scale expansion in GMP facilities
- NTF induction: Cytokine priming to enhance NTF secretion
- Quality testing: Multiple release criteria for identity, purity, potency
- Cryopreservation: Ready-to-use frozen doses
The manufacturing process is patented and represents a significant barrier to entry for competitors.
- ALS: Phase 3 completed; BLA submission under consideration
- AD: Phase 2 ongoing
- Orphan drug designation: Received for ALS in multiple jurisdictions
- Fast track designation: Granted by FDA for ALS
As a public company (NASDAQ: BCLI), BrainStorm has:
- Market cap: ~00M (as of 2024)
- Cash runway: Through 2025 based on public filings
- R&D expenses: ~0M annually
- No current revenue (clinical-stage)
BrainStorm is developing next-generation approaches:
- Allogeneic (off-the-shelf) MSC-NTF cells
- Enhanced NTF secretion through gene editing
- Combination approaches with small molecules
- Direct delivery to CNS targets
The AD program represents a significant opportunity:
- Large patient population
- Unmet medical need
- Potential for disease modification
- Companion biomarker development