Treatment of ALS based on transplantation of glial restricted progenitors

Johns Hopkins University, Baltimore MD

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Abstract

? DESCRIPTION (provided by applicant): 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.