Application of Glilal Progenitors in treatment of ALS

Project number:

STRATEGMED1/233209/12/NCBR/2015

Consortium members: 

• Mossakowski Medical Research Centre Polish Academy of Sciences
• Institute of Human Genetics 
• Clinical University Hospital in Olsztyn 
• VETREGEN

Sponsors: 

The National Centre for Research and Development

ABSTRACT:

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder with no cure. Patients who suffer from ALS typically die within 2-5 years of diagnosis. Recent progress in regenerative medicine has raised hope for a treatment breakthrough. The significant role of glia for the proper function of motor neurons has been recently reported, and efficient methods to isolate glial precursors have been established. Rodent models have shown that glial precursors of fetal origin display very high therapeutic potential, and are characterized by extensive engraftment and robust therapeutic effect. We propose to use fetal glial progenitors for the treatment of ALS. The therapeutic utility of cell delivered via the intraarterial or intrathecal route will be evaluated pre-clinically, followed by studies in spontaneous canine degenerative myelopathy. Allografting of progenitors in dogs is particularly attractive, as it will enable the optimization of therapeutic procedure in a clinically relevant setting, including utilization of catheter-based delivery, MRI cell tracking and to assess immunogenicity. Following the pre-clinical phase, the optimized therapeutic strategy will be used to treat ALS patients. The application of these latest developments in neurobiology, interventional neuroradiology, and regenerative medicine should result in a long-awaited cure for ALS.