Stem Cell Therapy

What It Is

Dr. Eva Feldman and Dr. Lisa McGinley of the NeuroNetwork for Emerging Therapies looking at an image from stem cell therapy research
Drs. Lisa McGinley & Eva Feldman

Stem cells are a cell type that serves as the source from which other bodily cells (e.g., neurons, skin cells) differentiate. Stem cells can be “totipotent,” meaning they can develop into any cell type, or “pluripotent,” meaning that they can develop into certain cell types.

Currently, neurodegenerative diseases like Alzheimer’s disease and ALS lack effective drug treatments. Researchers are testing if stem cell therapy helps slow neuron (nerve cell) deterioration.

Stem cell therapy has also emerged as a potential option for difficult-to-treat kidney diseases.

  • Fibroblast skin cells from a skin biopsy can be “reprogrammed” into stem cells. By wiping clean the cells’ original tissue-specific properties, they become induced pluripotent stem cells (iPSCs). iPSCs can then be converted into other cell types, such as neurons.
  • iPSCs are patient-specific, and contain the genetic profile of the patient.
  • In neurodegenerative disease research, these patient-specific iPSCs can be changed into neurons, microglia (neuron-supporting cells), or other cells involved in disease progression. This enables study of the disease on the patient’s specific genetic background.
  • Stem cells, following specific ethics guidelines, are being developed into cell therapies for neurodegenerative diseases, such as “NSI-566RSC” cells, a form of neural stem cells, for ALS clinical trials.