ALS: Mesenchymal Stem Cell Treatments in Rodents

What is ALS?

Many people have heard of amyotrophic lateral sclerosis, also known as ALS. It is a devastating neurodegenerative disease that affects strength and movement. ALS affects the neurons connect our brain to the rest of our body and control movement. In ALS, the neurons break down. While it can be genetic 10% of the time, 90% of the time it is spontaneous. The exact cause of the disease is unknown, which makes it difficult to treat.

 What are the treatment options?

Treatment normally includes includes physical therapy, speech therapy, social work, nursing, and other resources. There is only one medication that is formally approved to treat ALS, called Riluzole. Riluzole has been shown to slow the progression of ALS. It is neuroprotective, meaning it protects the neurons that are still there, and reduces the spasmodic qualities of the muscles affected by ALS. Research is being done on new treatment options.

The Science: Stem Cells

Stem cells are cells in many tissues of the body that have potential to become different types of cells. For example, a stem cell could become bone, skin, muscle, fat, nerves, etc. Of course, this means that scientists are especially interested in them because of this potential. They can be harvested from many different tissues in the body. At the institute, stem cells are harvested from umbilical cord tissue. They are then isolated and culture-expanded, meaning they are grown in the lab on special medium that help them grow and multiply. They are then injected into patients. This can be done intravenously or intrathecally, into the spinal canal. Many studies use similar techniques and are still in the early phases.

Stem Cells in Rodent Models

Study 1

Because scientists know one gene that causes ALS, many animal studies target that gene. That gene is called SOD1. Scientists cause the gene mutation in mice. They then harvested mesenchymal stem cells (MSCs), grew them in the lab, and then injected them into the mice. They found that motor neuron loss in these mice was slower. They also found that if the MSCs lasted long enough in the body, they were able to migrate to the brain and spinal cord and begin to make new tissue there. This, however, was rare. Overall, this study concluded that MSC transplantation increased neuronal survival and prevented inflammation, but it was less likely to replace lost neurons.

Study 2

Another study in genetically modified mice showed a therapeutic role. This study found that mice that had just started to present with ALS symptoms improved symptomatically when given MSCs. This study found that their survival also improved and the disease progression was delayed, similar to the last study. However, this study found that only a few MSCs migrated to the brain and spinal cord.

Study 3

Lastly, another study found that the site of administration mattered. They administered MSCs intrathecally, meaning they injected the cells into the spinal cord. There, the cells mixed with the cerebrospinal fluid, the fluid that surrounds the spinal cord and brain. They found that injected the MSCs into the spinal cord increased their lifespan, meaning that the cells themselves lasted longer. This also helped them protect motor neurons, which improved motor performance in the genetically modified mice. Like the studies above, they found that MSCs had an anti-inflammatory property. This study injected MSCs multiple times, at 8, 10, and 12 weeks of age. Overall, they found that it delayed disease onset, prolonged lifespan of the mice, improved their motor function, and preserved existing motor neurons.

Miscellaneous studies

There are many, many studies that examine stem cells in ALS. They have found that the effects were dose-dependent, meaning that more MSCs increased the effect. They found that certain types of stem cells, particularly those from fat, were more likely to end up in the central nervous system, but less likely to turn into certain types of neurons. They were, however, more likely to protect existing cells.

How does this translate to patients?

As stated above, research is still ongoing. There are some clinical trials that are being done in humans. Many of them have already confirmed the safety of MSC transplants in humans. The research that is being done now is to evaluate the symptomatic effects of these transplants. Based on the mice models, the researchers have high hopes that these studies will help ALS patients.


In summary, stem cells show promising potential to treat ALS. Many studies are still in the first phase and are only looking at animal models. However, the initial results are promising and the field is ever evolving.

Stem cell use for ALS is an emerging field. At the Stem Cells Transplant Institute, we have developed state-of-the-art treatment protocols for ALS patients in culture expanded cells. If you are interested in learning more about how you can use this technology for your own health goals, please contact our institute today.