Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder without a cure. Patients who suffer from ALS typically die within two-to-five years of diagnosis. Recent progress in regenerative medicine has raised hope for a breakthrough. The significant role of glia for the proper function of motor neurons has been recently reported, and efficient methods to isolate glial-restricted precursors (GRP) have been established. It has been shown in rodent models that GRPs of fetal origin display the highest therapeutic potential among all other sources, because they are characterized by extensive engraftment, differentiation, and robust therapeutic effect. In this project, we propose to use fetl GRPs for the treatment of ALS. The Allografting of GRPs in pigs is particularly attractive, as it will be performed in a clinically relevant setting, including utilization of catheter-based cell delivery, with a clinical MR scanner for cell tracking and assessment of immunogenicity/immunoprotection. The application of the latest developments in neurobiology, interventional neuroradiology, and regenerative medicine should result in a long-awaited cure for ALS.

Public Health Relevance

Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive neurodegenerative disease, with most patients dying within three-to-five years of diagnosis and effective treatment not available. The application of stem/progenitor cells offers the greatest potential for restoration of lost neurological function. Accordingly, we propose to focus our project on regenerative medicine and the application of glial progenitor cells for the treatment of ALS.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Research Project (R01)
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Clinical Neuroscience and Neurodegeneration Study Section (CNN)
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Gubitz, Amelie
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Johns Hopkins University
Schools of Medicine
United States
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