Modified Stem Cells for Treating Neurodegenerative Diseases

Emory University
posted on 07/07/2009

Emory University is looking for companies interested in commercializing a stem cell therapy with the potential to treat Parkinson's or Alzheimer's disease. This technology involves transplanting human non tumor-derived, neuronal progenitor cells capable of developing into adult neurons. These cells can be modified via gene therapy prior to transplant and tailored to perform a selected function. This therapy provides a new approach for treating neurodegenerative diseases.

Applications

Treatment of degenerative neurological disorders including Parkinson's and Alzheimer's disease.

Highlights

Uses adult human neurogenic stem cells rather than human embryo cells.
Cells can be modified to perform specific function or exhibit certain characteristics via gene therapy techniques.

Technical Summary

Neurological disorders such as Parkinson's disease (PD) and Alzheimer's disease (AD) are associated with the loss of specific neurons in the brain accompanied by alterations in behavior and/or cognitive function. Previous cell-based therapies aimed at treating AD & PD have employed transplantation of genetically altered fibroblasts (collagen producing cells) or fetal tissue and met with limited success.

Researchers at Emory have developed a possible method for treating neurodegenerative diseases using human neuronal progenitor cells harvested from the subventricular zone of the brain. These cells have not only been shown to develop into adult neurons but also can be modified through common gene therapy techniques to perform a specific function or exhibit a specific characteristic (such as expressing a growth factor). Once modified, the cells are transplanted back into the area of the brain that has degenerated, replacing the lost neurons. This technology provides a tunable cell-based therapy with the potential for restoring/rescuing neuronal function affected in a variety of neurological disorders.