BrainStorm Cell Therapeutics Inc. a leading developer of adult stem cell technologies and therapeutics, announced that intramuscular transplantation of autologous, astrocyte-like cells that produce and secrete Neuro-Trophic Factors (NTFs), representing the company’s NurOwn technology platform, preserved motor function, significantly inhibited the degeneration of the Neuro-Muscular Junctions (NMJs), and preserved the myelinated motor axons in an animal sciatic nerve injury model.
“The findings from this study demonstrating that BrainStorm’s autologous NurOwn stem cell therapy can alleviate signs of sciatic nerve injury is an important milestone for the company,” said Chaim Lebovits, president of BrainStorm. “One of the major caveats of stem cell transplantation is the fate of the transplanted cells. In the current study, we show that our transplanted cells can integrate and survive in the host muscles of animals after sciatic nerve crush for at least 3 weeks. This preclinical work provides additional support for the upcoming phase I clinical trial of NurOwn for patients with Amyotrophic Lateral Sclerosis (ALS) and other neurological disorders.”
In a study conducted at Tel Aviv University, Mesenchymal Stem Cells (MSCs) isolated from the femurs and tibias of adult rats were developed into NurOwn using a two-step medium based differentiating protocol to induce the MSCs into NTF secreting cells. These cells produce and release high amounts of NTFs, such as Glial Derived Neurotrophic Factor (GDNF) and Brain Derived Neurotrophic factor (BDNF). The NTF secreting cells (NurOwn) were labelled with Super-Paramagnetic Iron Oxide (SPIO) to enable tracking of surviving cells following injection into the muscles of the right hind limb 24-hours after sciatic nerve crush.
Four days after transplantation, there was a statistically significant beneficial effect on the motor function in the NurOwn treated animals compared to the control rats, which did not receive cell transplants, or rats transplanted with non-differentiated MSCs. The high compound muscle action potential and low latency indices recorded in the hind limb muscles of NurOwn treated animals provided evidence that NurOwn preserved the myelinated motor axons and innervated peripheral muscles. Histology of the animal’s hind limb muscles 3-weeks after transplantation revealed significant amount of pre-labelled NurOwn cells and high levels of BDNF in the muscles.
One of the most common peripheral neuropathy is sciatica, damage to the sciatic nerve, with a high reported prevalence. Sciatica is characterized by muscle weakness, reflex changes and numbness. Tumours, cysts or other extra spinal insults can cause sciatica. The majority of sciatica patients suffer from persistent and severe types of pain, motor dysfunctions and prolonged disability.
Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig's Disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. According to the ALS Association, approximately 5,600 people in the US are diagnosed with ALS each year and it is estimated that as many as 30,000 Americans may have the disease at any given time and the financial cost is exceedingly high, and it is estimated that in the advanced stage, care can cost an average of $200,000 per year, which represents more than a $6 billion cost to the healthcare system.
BrainStorm’s core technology, NurOwn, is based on the scientific achievements of Professor Eldad Melamed, former Head of Neurology, Rabin Medical Centre, and Tel-Aviv University, and a member of the Scientific Committee of the Michael J Fox Foundation for Parkinson's Research, and Professor Daniel Offen, Head of the Neuroscience Laboratory, Felsenstein Medical Research Centre (FMRC) at the Tel-Aviv University.
The NurOwn technology processes adult human mesenchymal stem cells that are present in bone marrow and are capable of self-renewal as well as differentiation into many other cell types. The research team is among the first to have successfully achieved the in vitro differentiation of adult bone marrow cells (animal and human) into astrocyte-like cells capable of releasing neurotrophic factors, including Glial-Derived Neurotrophic Factor (GDNF). The ability to induce differentiation into astrocyte-like cells along with intramuscular or intrathecal (or other) delivery makes NurOwn technology highly attractive for treating ALS and Parkinson’s disease as well as MS and spinal cord injury.
BrainStorm’s stem cell therapy contains human mesenchymal stromal cells induced to differentiate into astrocyte-like cells secreting neurotrophic factors by means of a specific differentiation-inducing culture medium.
BrainStorm Cell Therapeutics Inc. is an emerging company developing adult stem cell therapeutic products, derived from autologous (self) bone marrow cells, for the treatment of neurodegenerative diseases.