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.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Research Project (R01)
Project #
5R01NS091110-05
Application #
9700730
Study Section
Clinical Neuroscience and Neurodegeneration Study Section (CNN)
Program Officer
Gubitz, Amelie
Project Start
2015-09-15
Project End
2020-06-30
Budget Start
2019-07-01
Budget End
2020-06-30
Support Year
5
Fiscal Year
2019
Total Cost
Indirect Cost
Name
Johns Hopkins University
Department
Radiation-Diagnostic/Oncology
Type
Schools of Medicine
DUNS #
001910777
City
Baltimore
State
MD
Country
United States
Zip Code
21205
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