A renewable, engineered cell product shows potential for treating neurodegenerative and retinal diseases

Newswise —

The fundamental reason for a range of debilitating conditions known as neurodegenerative diseases, such as ALS, Parkinson's disease, and retinitis pigmentosa, is a gradual reduction in neurons. Although methods to generate or substitute damaged neurons with stem cells are being researched, a newly published study in Stem Cell Reports proposes safeguarding current neurons as a substitute treatment.

Scientists at Cedars-Sinai Medical Center in the United States have utilized genetic engineering to create neural progenitor cells (NPCs), which are responsible for generating neurons and astrocytes. These cells were modified to release a specific neuroprotective protein and tested in animal models of neurodegenerative disease. The research builds on a clinical trial that demonstrated the safety and potential efficacy of the approach in treating ALS. The team, led by Clive Svendsen, created potentially patient-specific NPCs by generating engineered cells from reprogrammed adult human blood cells. The engineered NPCs successfully prevented the death of neurons in a rat model of ALS and photoreceptor cells in rats with retinal disease. The transplanted cells remained viable for up to nine months in the rat spinal cord, where they continued to release the neuroprotective protein and developed into neuron-supporting astrocytes, which may be responsible for the observed therapeutic effects.

The findings of this study offer compelling evidence and justification for future clinical trials examining the safety and effectiveness of using engineered, long-lasting, and patient-specific NPCs for the treatment of neurodegenerative diseases.