NurOwn® is a cell therapy platform that builds on the enormous research effort that has gone into developing mesenchymal stem cells (MSCs) as a treatment for human diseases. BrainStorm has taken MSCs a step further in developing a therapy customized for the treatment of neurodegenerative diseases.
Mesenchymal Stem Cells
MSCs are multipotent precursor cells found in many tissues, but most commonly obtained from the bone marrow, adipocytes or placenta. MSCs have been used in human studies since the 1990s, initially to enhance engraftment of bone marrow transplants, but eventually researchers discovered that MSCs are able to tamp down the immune system in a variety of different ways.
This led to their being studied as treatments for a variety of immune and inflammatory diseases, such as graft-versus-host disease, inflammatory bowel disease, rheumatoid arthritis and multiple sclerosis, and have been studied in cardiovascular disease, stroke, kidney disease, and many other areas – indeed there have been hundreds of clinical trials completed or ongoing for MSCs in various conditions. Prochymal® (remestemcel-L) is an MSC product that received conditional approval for the treatment of children with acute, steroid-refractory graft versus host disease in Canada and New Zealand.
How NurOwn® Makes a Better Cell
Where others leave off with MSCs, BrainStorm begins. Our patented NurOwn® technology takes MSCs and, by growing them in proprietary conditions, converts them into biological factories secreting a variety of neurotrophic factors (NTFs). NTFs are growth factors known to support the survival of neurons in a variety of conditions, and in animal models of many neurodegenerative diseases.
The NurOwn® technology was developed in the laboratories of Professor Dani Offen and the late Professor Eldad Melamed, at Tel Aviv University, and has been the subject of many publications. Our NTF-secreting MSCs (or, MSC-NTF cells) are designed specifically to treat neurodegenerative diseases by allowing us to deliver several NTFs at or close to the site of injury.
MSC-NTF cells also possess immunomodulatory characteristics and in some diseases, this may provide another mechanism for the cells providing a benefit. MSC-NTF cells secrete a variety of NTFs including Glial Derived Neurotrophic Factor (GDNF), Brain Derived Neurotrophic Factor (BDNF), Vascular Endothelial Growth Factor (VEGF), and Hepatocyte Growth Factor (HGF). Our cells can protect neurons from toxins and have been effective in animal models of ALS, sciatic nerve injury, Parkinson’s disease, multiple sclerosis, optic nerve ischemia and Huntington’s disease.
Over 60 patients with amyotrophic lateral sclerosis (ALS) have been treated with NurOwn® in clinical trials conducted in the United States and Israel, establishing an excellent safety and tolerability profile and demonstrating promising signs of efficacy.