Neurodegenerative diseases are disorders characterized by the progressive degeneration and death of nerve cells (neurons) in the brain and/or spinal cord. This degeneration leads to a decline in cognitive, motor, and functional abilities, resulting in conditions such as Alzheimer’s disease and amyotrophic lateral sclerosis (ALS). These diseases pose significant challenges as they affect essential neurological functions, leading to severe disability and a diminished quality of life. They are particularly problematic due to their complex pathologies, involving multiple cellular and molecular pathways, which complicate the development of effective treatments. Furthermore, the brain’s limited capacity for self-repair and regeneration exacerbates the difficulty of halting or reversing the progression of these diseases.  

• In the United States, nearly 7 million individuals are living with a neurodegenerative disease. 

• Most neurodegenerative diseases currently lack effective treatment options. 

Stem cells are special types of cells that can turn into different types of cells in the body, including nerve cells. They can be “totipotent,” meaning they can differentiate into any cell type, or “pluripotent,” meaning that they can develop only into certain cell types. 

• Skin cells (fibroblasts) from a skin biopsy can be grown in laboratory culture dishes and “reprogrammed” into stem cells. By wiping clean the cells’ original tissue-specific identity, they become induced pluripotent stem cells (iPSCs). iPSCs can then be converted into other cell types.
• iPSCs are patient-specific, meaning they contain the genetic profile of the patient they are derived from. Patient-specific iPSCs can be changed into neurons, glia (neuron-supporting cells), or other cells involved in neurodegenerative disease progression. This enables researchers to study the disease on the patient’s specific genetic background.

Stem cells are a promising treatment for neurodegenerative diseases since they can potentially replace the nerve cells that have been lost or damaged. By introducing stem cells into the affected areas of the brain or spinal cord, we might be able to restore some of the lost functions, slow down the progression of the disease, and possibly even repair some of the damage. Additionally, scientists can use stem cells to create models of these diseases in the lab, helping them to better understand how the diseases work and to test new treatments. Within the NeuroNetwork for Emerging Therapies, we are committed to developing novel stem cell-based therapies to treat neurodegenerative diseases, such as Alzheimer’s disease and ALS.