BrainStorm Cell Therapeutics (NASDAQ:BCLI) is a biotechnology company developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as
- Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease and Motor Neuron Disease)
Our platform technology, NurOwn®, uses proprietary culture conditions to induce mesenchymal stem cells (MSCs) to secrete high levels of neurotrophic factors (NTFs) known to promote the survival of neurons. Our research efforts have shown that these MSC-NTF cells might be an effective tool for battling neurodegenerative diseases.
- That delivery of these neurotrophic factors to injured or degenerating neurons can prolong their survival and improve their function.
That MSCs, which possess beneficial properties including the ability to home to sites of injury and to dampen immune responses, represent the ideal biological delivery system for neurotrophic factors.
MSC-NTF cells have been shown in the laboratory and in animal models of neurodegenerative diseases to have a beneficial effect, and BrainStorm and our academic collaborators have published over 20 papers in leading scientific journals demonstrating NurOwn’s mechanism of action and efficacy. We invite you to view the Technology and Pipeline areas of our website to learn more.
NurOwn® in the Clinic
Human testing of MSC-NTF cells was initiated 2010 in ALS patients, a program that has now advanced to phase 2 clinical trials in the United States and Israel. We have completed two single-arm clinical trials at Hadassah Medical Center in Jerusalem, in which a total of 26 ALS patients received MSC-NTF cells. These studies established the safety profile of the cells and provided indications of a treatment benefit.
We recently completed a 48 patient randomized, double-blind, placebo-controlled phase 2 study of MSC-NTF cells at three prestigious academic medical centers in the United States.
NurOwn® has received Fast Track status from the US Food and Drug Administration (FDA) in ALS, and has additionally been granted Orphan Status for ALS by the FDA and the European Medicines Agency (EMA).
BrainStorm has developed a proprietary, stem cell based technology that delivers growth factors that can help neurons live longer at or near the site of injury or damage. We are developing this innovative therapy for the treatment of various neurodegenerative diseases. It has been tested in several animal models for various diseases, and has shown safety and promising signs of efficacy in clinical trials for amyotrophic lateral sclerosis.
Neurodegenerative Diseases and Stem Cell Research
Neurodegenerative diseases such as amyotrophic lateral sclerosis, Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, multiple sclerosis, and many others afflict millions of people around the globe, but for the most part lack effective therapies. The need for therapies that can replace or repair dead, dying, stressed or damaged neurons has naturally led investigators to stem cell-based therapies.
In general, there are two distinct and potentially complimentary approaches to treating neurodegenerative diseases with cellular therapies – stem cells can be used to replace dead, damaged or dying neurons, or can be used to support damaged or dying neurons, through a variety of mechanisms including quieting the immune system and inflammation, secretion of growth factors, and differentiation into glial cells.
The first approach presents some important technical challenges, particularly in neurology – not only must one deliver neurons or cells that will turn into neurons, but one must insure those neurons are in the right place and making the right connections with other neurons in the central and/or peripheral nervous system, or with other tissues like muscle. Mesenchymal stem cells sclerosis, also called mesenchymal stromal cells, or MSCs, are an example of the second approach and have been used by researchers to treat a variety of conditions, including some neurodegenerative diseases.
We have made a major advancement on the use of mesenchymal stem cells with our NurOwn® technology platform.
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.
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