Stem Cells I NeuroNetwork
Neurosurgeon and neurologist Dr. Kevin Chen looking at a microscope

Using stem cells to combat neurodegenerative diseases.

The Problem

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.

brain stem blue green slice
Stem cells (green) that have been transplanted into an animal model brain

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.  

ALS preclinical studies
Launch preclinical studies of stem cells as a potential ALS therapy, testing feasibility in animal models.
stem cells stem cells
ALS Phase 1
Report on phase I trial of stem cells in ALS, the first findings in humans, which deemed stem cells safe. Plans are set in motion for a phase II trial.
a neurosurgeon transplanting stem cells into the spinal cord of an ALS patient a neurosurgeon transplanting stem cells into the spinal cord of an ALS patient
Early successes in Alzheimer's disease
Successfully transplant stem cells producing the hormone insulin-like growth factor, which stimulates metabolism and growth, into the brains of Alzheimer’s disease mice. (photo shows stem cells (dark) transplanted into an animal model)
stem cells shown as dark spots that have been transplanted into an animal model brain stem cells shown as dark spots that have been transplanted into an animal model brain
More discovery
We continue to develop and advance our stem cell program, discovering stem cells expressing insulin-like growth factor I transplanted into brains of Alzheimer’s disease mice enhances memory. (photo shows colored imaging of stem cells transplanted into animal model brains)
colored imaging of stem cells transplanted into animal model brains colored imaging of stem cells transplanted into animal model brains
Stem cell survival
We use magnetic resonance imaging (MRI) to track movement of transplanted stem cells in brains of animal models and show stem cells survive in brains for as long as ten weeks. (photo shows stem cells (green) that have been transplanted into an animal model brain)
stem cells (green) that have been transplanted into a preclinical model brain (blue). stem cells (green) that have been transplanted into a preclinical model brain (blue).
Mini brains
Collaborated with U-M bioengineering colleagues to artificially grow “mini brains” from induced pluripotent stem cells. (photo shows a brain organoid)
Understanding nerve health
Harnessing power of stem cells to understand how nerve and supporting cells (Schwann cells) impact nerve health and metabolism.
brain organoid grown in a lab brain organoid grown in a lab
Stem Cell Therapy Initiatives
Interneurons

Specialized nerve cells in the central nervous system act as connectors between sensory and motor neurons, playing a crucial role in processing information and coordinating responses within the brain and spinal cord.

Mini Brains

Also known as brain organoids, mini-brains are tiny, three-dimensional structures grown from stem cells in cell culture that mimic some features of a real brain and offer exciting research possibilities.

Stem Cell Therapy

Looking into stem cells as treatments for neurologic diseases.