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.
"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.
Make of that what you will; the good news I take away is that a variety of methods for attacking this class of neurodegenerative disease are currently under investigation. Diversity of study is always promising in the long term, and any progress towards curing or preventing neurodegeneration is a good thing. In the near future, probably within 10 years, you'll be able to replace failing organs with engineered tissue should your personal worst case medical scenario come to pass - but age-related damage to the brain has to be repaired in situ. Hence those of us interested in healthy life extension should also be interested in neuroregenerative work.