What are Neural Stem Cells?

The importance of neural stem cells and progenitor cells in regenerative medicine

The human nervous system is the command center of the body, controlling movements and responses to the environment from signals that originate in the brain.  The nervous system is extremely complex, and guides everything you do, whether you think about it or not. Diseases, injury, and aging can considerably damage the nervous system, with limited ability to heal and self-repair.   

Due to the irreversible damage seen with severe neurodegenerative diseases and injuries to the spinal cord or brain, it was once thought that the body was incapable of making new neural cells.  Researchers now know that neural cells can be regenerated by stem cells in the brain through a process called neurogenesis.  While neural stem cells are predominantly found in the embryonic brain and active during development, some neural stem cells persist in specific regions of the adult brain. 

Neural stem cell research inspires hope among scientists and patients for improved treatment of an array of neural diseases and injuries. 

What are neural stem cells?

Neural stem cells (NSCs) are multipotent stem cells that are committed to the neural fate.  Neural stem cells can differentiate into the specific functional cells of the nervous system, including neurons (responsible for conducting electrical signals), oligodendrocytes (cells that insulate neurons to improve electrical transport), and astrocytes (cells that have many functions, including regulating energy use, reacting to injury, and protecting against infection).  Neural stem cells have some capacity to self-renew in the body and in culture. 

Neural progenitor cells are descendants of neural stem cells, which cannot self-renew and have limited abilities to divide and proliferate.  Neural progenitor populations can have varying degrees of potency, with the ability to differentiate into one or more cell type as unipotent, bipotent, or multipotent cells.  

Neural precursor cells (NPCs) is a general term referring to a mixed population of both neural stem and progenitor cells.  Neural stem and progenitor cells can be cultured in the lab as either adherent cells in a monolayer culture or as neurospheres in suspension, depending on the culture system employed.   

Helpful tip:  The term “neural” refers to the broad group of cells that make up the brain and nerves.  “Neuronal” is a more specific term, referring to neurons in particular. 

How can stem cells help treat neural diseases? 

Neural stem cells (NSCs) are mostly dormant in the adult body, but there is ample evidence of their importance in plasticity, aging, disease, and regeneration of the nervous system.  The small number of resident NSCs in adult tissue has been a limiting factor for clinical applications, but NSCs and NPCs derived from human induced pluripotent stem cells (hiPSCs) hold great promise in regenerative medicine.  These cells are particularly useful for neurological disease modeling to study neurodevelopment and neurodegenerative diseases, as well as for future cell-based therapies.  

Translational medicine and cell transplantation using neural stem and progenitor cells holds great promise to treat a range of diseases and injuries, such as Alzheimer’s disease, dementia, Parkinson’s disease, multiple sclerosis, as well as brain and spinal cord injuries. 

Key applications for neural stem cells in the lab 

• Disease modeling essentially includes studying diseased cells or the progression of a disease in a dish in order to understand characteristics of healthy and diseased cells and the functional mechanisms of a disease.  Ultimately this intricate understanding is used to develop new therapies and treatments. 
• Pharmaceutical testing on functional human cells in culture is essential to understand how effective new drugs intended to treat neural diseases may be, as well as to learn potential side effects and reactions to the drug on the cellular level. Prior to clinical trials, is important to first test the effects of these compounds in neural cells in a dish before treating a person.    
• Regenerative medicine is a field of research with a focus of replacing or supplementing cells and tissues that have been damaged by disease or injury with new cells or cell products. Many researchers, institutes, and biotechnology companies are focused on exploring the potential of creating cell-based therapies using iPSC-derived neural cells and other engineered cell types to treat neurodegenerative diseases. 

Examples of neural regenerative medicine research groups

• The Goldman Lab, University of Rochester Medical Center – The focus of the Goldman lab’s research in the Center for Translational Neuromedicine is to understand the regulatory control of stem and progenitor cells in the central nervous system, and to build upon that knowledge to design new ways to treat neurological diseases through cell and gene therapy techniques.  The lab uses mammalian and songbird models to study neurogenesis in the adult brain, with the goal of developing ways to induce neuronal production in adults as a means of repair to damaged or diseased neural circuits.

• The Gage Lab, the Salk Institute – One of the Gage lab’s key areas of focus is developing cellular models to understand the biological aspects of complex neuropsychiatric and neurodevelopmental diseases, such as Schizophrenia, Autism Spectrum Disorders, Bipolar Disease, Multiple Sclerosis, Parkison’s Disease, and others. The lab reprograms adult cells from patients with a neurodegenerative disease to create unique iPSC lines that express that person’s disease genotype.  The iPSCs are then differentiated into neurons, which the lab studies to better understand the cellular development, disease phenotypes, and differences in neuronal function between diseased and non-diseased tissue.  This groundbreaking research will someday lead us to new diagnostic tools and critical therapeutic treatments.

• Neural Stem Cell Institute (NSCI) – NSCI is a non-profit stem cell research institute dedicated to developing regenerative therapies for the central nervous system (brain, spinal cord, and retina).  Scientists at NSCI study how to activate neural stem cells in the body to aid in injury repair, generate cell models from patient-derived iPSCs to study diseases such as macular degeneration and Alzheimer’s Disease, and conduct research the specific neural stem cells in the eye (retinal pigment epithelial stem cells) to advance the treatment of blinding disorders. NSCI also houses a core facility to support other research laboratories by providing unique stem cells, tools, and expert services for a wide range of regenerative medicine applications.

• Neurona Therapeutics – Neurona Therapeutics is a biotherapeutic company with clinical-stage technologies focused on discovering and developing neural cell therapies to treat chronic diseases of the nervous system. The company’s regenerative medicine platform includes the methods to produce differentiated and functional neuronal cells for targeted repair of a damaged nervous system. A list of the various clinical trials sponsored by Neurona Therapeutics’ can be found on clinicaltrials.gov.

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