New York - BrainStorm Cell Therapeutics, today announced a breakthrough in the development of adult stem cell therapeutics for neurodegenerative diseases. The company's scientific collaborators at Tel Aviv University, have successfully used adult stem cells to produce glial derived neurotrophic factor (GDNF). GDNF producing cells hold great promise for treatment of Parkinson's and other neurodegenerative diseases.
In the current study, the research team at Tel Aviv University used proprietary technology, to differentiate human bone-marrow-derived mesenchymal-stem cells into neural supporting cells that produce GDNF. Worldwide rights to these research results are exclusively licensed to BrainStorm.
GDNF has been shown to protect dopaminergic brain cells, neurons that produce dopamine that becomes depleted in Parkinson's patients. GDNF has also been shown to have a beneficial role in protecting neurons in animal models of Amyotrophic Lateral Sclerosis (ALS) and Spinal Cord Injury (SCI).
Most of the current treatment of neurodegenerative diseases provide limited benefit to patients. Drugs for Parkinson's disease, which focus on dopamine supplementation, often cause prohibitive side effects. To overcome these problems, researchers around the world are intensively exploring novel cell- and gene-based therapies for replacement and augmentation of the lost neurons. Prominent among these novel therapies are attempts to deliver GDNF to the site of neuronal regeneration.
In the current BrainStorm-sponsored study at Tel Aviv University, biochemical and immunological methodology showed that human bone marrow mesenchymal stem cells may be uniquely differentiated into cells that resemble astrocytes, express astrocytic markers and produce significant levels of GDNF.
The same scientific team at Tel Aviv University, led by Professor Eldad Melamed and Dr. Daniel Offen, have previously developed patent-pending technology to differentiate human bone marrow into dopamine producing neuron-like cells, which showed functional benefit in animal models of Parkinson's disease. This technology is also exclusively licensed to BrainStorm.
"Neurologists have long thought that GDNF can be used to preserve and maintain the integrity of dopaminergic neurons in Parkinson's disease. However, delivery to the appropriate location is a major challenge. Direct delivery of the protein has failed and there are current ongoing attempts at gene therapy. We believe that our approach, based on neural transplantation of stem cell derived GDNF producing cells, without any genetic manipulation, is preferable," said Prof. Eldad Melamed, Chairman of the Scientific Advisory Board.
In addition, "The use of a patient's own bone marrow stem cells to generate neural cells for replacement and support of a patient's damaged dopaminergic cells is a strategy that will avoid problems of immunological rejection," said Dr. Daniel Offen, Chief Scientist.
"This is a remarkable achievement which combined with our previous success in producing dopaminergic cells, gives us a two-pronged, synergistic approach to develop long term alternatives for the treatment of Parkinson's disease and other debilitating disorders, such as ALS or SPI," said Yaffa Beck, President and CEO. " We look forward to demonstrating benefit of the GDNF producing cells in animal models and are intent on developing both our dopaminergic and GDNF differentiation processes to the clinic," she added.
SOURCE: BrainStorm Cell Therapeutics Inc.