Brainstorm Cell Therapeutics Inc. is a biotechnology company focused on the development and commercialization of autologous cellular therapies for neurodegenerative diseases. The company’s primary asset is NurOwn®, a proprietary technology that engineers a patient’s own bone marrow-derived mesenchymal stem cells to secrete high levels of neurotrophic factors. NurOwn® is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS), progressive multiple sclerosis (PMS), and Alzheimer’s disease (AD), with ALS representing the company’s highest priority indication. The company operates primarily through its U. S. and Israeli subsidiaries, conducting research, manufacturing, and clinical development activities across these locations.
Brainstorm Cell Therapeutics Inc. does not currently generate revenue from product sales, as NurOwn® remains an investigational therapy and has not received regulatory approval for commercial sale. The company’s financial resources have historically been derived from a combination of non-dilutive funding sources, including government grants, nonprofit awards, and milestone-based payments. Notably, the company received a $15,912,390 grant from the California Institute for Regenerative Medicine (CIRM) to support its pivotal Phase 3 ALS trial, with full disbursement completed by December 2020. Additional funding has come from the National Multiple Sclerosis Society, The ALS Association, and I AM ALS, supporting biomarker research and clinical development in PMS and ALS. The company has also received gross proceeds from treatment programs under regulatory pathways such as the Israel Hospital Exemption, though these did not cover full trial costs.
The company operates through the following segments:
- NurOwn® Autologous Cellular Therapy: This segment focuses on the development of NurOwn® (MSC-NTF cells) as an investigational treatment for neurodegenerative diseases. The technology involves harvesting a patient’s bone marrow, isolating and expanding mesenchymal stem cells, cryopreserving them, differentiating them into neurotrophic factor-secreting cells, and administering them intrathecally via lumbar puncture. NurOwn® has been granted Fast Track designation by the U. S. Food and Drug Administration (FDA) for ALS and Orphan Drug status by both the FDA and the European Medicines Agency (EMA) for this indication. The company has completed Phase 3 clinical trials in ALS and Phase 2 trials in PMS, with ongoing efforts to align with the FDA on a Special Protocol Assessment (SPA) for a Phase 3b registrational trial in ALS.
- Exosome-Based Platform Technology: This segment involves research and development of exosomes derived from NurOwn® (MSC-NTF cells) for potential therapeutic applications. These extracellular nano-vesicles are being studied for their ability to cross the blood-brain barrier, deliver bioactive molecules, and modulate inflammation in neurodegenerative diseases, acute respiratory distress syndrome (ARDS), and neurodegenerative eye disease. Research efforts include optimizing large-scale bioreactor culture, developing scalable GMP purification methods, characterizing exosome cargo and potency, and assessing stability and modification techniques. Preclinical studies have demonstrated statistically significant improvements in lung parameters in ARDS models when using NurOwn®-derived exosomes compared to exosomes from naïve mesenchymal stem cells.
Brainstorm Cell Therapeutics Inc. operates in the highly competitive biotechnology sector focused on regenerative medicine and neurodegenerative disease therapies. The company differentiates itself through its autologous cell therapy approach, which uses the patient’s own cells to minimize immune rejection and avoid the need for immunosuppressive drugs. NurOwn®’s mechanism of action—secreting multiple neurotrophic factors and modulating neuroinflammation—targets several pathways simultaneously, contrasting with many single-target therapies in development. While companies such as Corestem (South Korea), Kadimastem (Israel), and Ionis/Biogen are advancing alternative ALS treatments, Brainstorm’s NurOwn® has received regulatory incentives like Fast Track and Orphan Drug designations, supporting its path toward potential approval. The company’s intellectual property portfolio, including approximately 30 granted patents and expanding exosome-related applications, further strengthens its competitive position in the United States, Europe, Japan, Canada, and Israel.
Brainstorm Cell Therapeutics Inc. serves patients suffering from neurodegenerative diseases, particularly those with amyotrophic lateral sclerosis (ALS), progressive multiple sclerosis (PMS), and Alzheimer’s disease (AD). The company’s clinical trials have enrolled participants from leading medical centers in the United States, including Massachusetts General Hospital, Memorial Hospital in Worcester, Mayo Clinic, Dana Farber Cancer Institute, and City of Hope. In Israel, trials have been conducted at Hadassah Medical Center in Jerusalem. The company has also expanded access to NurOwn® through its Expanded Access Program (EAP), which has provided treatment to ALS patients who completed Phase 3 trial requirements at centers such as University of California, Irvine, Cedars-Sinai Medical Center, California Pacific Medical Center, and the University of Massachusetts Medical School. Additionally, the company has treated ALS patients under Israel’s Hospital Exemption pathway in collaboration with Sourasky Medical Center.