Stem cells are a potential treatment for ALS currently being studied in clinical trials. This treatment is not FDA-approved at this time, and the results of these trials will determine whether it ever becomes approved.
Amyotrophic lateral sclerosis (ALS) is a rare neurological disease of nerve cells involved in muscle movement. As these cells die, it results in gradually worsening muscle weakness and wasting which eventually leads to paralysis.
There’s no cure for ALS at this time. Current treatments instead focus on managing symptoms, promoting independence, and improving quality of life.
Doctors and scientists continue to investigate new ways to treat ALS. Stem cell treatments are one type of treatment that’s being studied.
Keep reading to learn more about stem cell treatments for ALS and where they are in development.
Stem cells are a type of cell that has the ability to become many different cell types. This is called differentiation. For example, stem cells in the bone marrow can go on to differentiate into specific types of blood cells.
Stem cells can also self-renew, unlike many cell types in the body. When a stem cell divides, the two resulting cells can be:
- two stem cells
- one stem cell and one differentiated cell
- two differentiated cells
There are two general types of stem cells:
Pluripotent stem cells
Pluripotent stem cells can become any type of cell in the body. This type of stem cell is found in human embryos.
However, in 2006, scientists developed a way to reprogram some types of adult cells to become pluripotent stem cells. These stem cells are called induced pluripotent stem cells.
Adult stem cells
Adult stem cells exist in various tissues and are involved in maintenance and repair. They work to replace cells that are lost through typical wear and tear, injury, or disease.
These stem cells are tissue-specific. This means that, unlike pluripotent stem cells, they’re more specialized.
For example, while adult stem cells in your intestines can replace intestinal cells that have been lost or damaged, they cannot go on to become skin cells or blood cells.
In ALS, a neurological disease, nerve cells that control muscle movements, called motor neurons, become damaged and die. Unlike cells in other parts of the body, like the skin and gut, nerve cells cannot regenerate after they’re damaged or die.
Additionally, we’re still learning more and more about the ALS disease process. What we do know is that ALS is complex, with many different factors that contribute to it. All of this makes identifying therapeutic targets more difficult.
Stem cells are an attractive treatment for ALS due to their regenerative effects and ability to differentiate into different cell types. The main ways that stem cells may be used to treat ALS are:
- using stem cells to deliver factors that work to bolster the health and survival of motor neurons
- replacing damaged or dead neurons with healthy neurons generated from stem cells
A 2023 review notes that many types of stem cells have been used in ALS preclinical research as well as in clinical trials:
- Mesenchymal stem cells (MSCs), the
most commonstem cell type used in ALS research, are a type of adult stem cell that can be isolated from many areas, such as bone marrow, muscle, and fat tissue.
- Hematopoietic stem cells are a type of adult stem cell isolated from the blood or bone marrow.
- Neural stem cells are isolated from embryonic tissue.
- Induced pluripotent stem cells are generated in a lab using adult cells.
Research into stem cell treatments for ALS
Now, let’s review a couple of clinical trials on stem cell treatments for ALS. Keep in mind that this is only a snapshot of research into the use of stem cells for treating ALS.
This trial was mainly concerned with the safety of the treatment. It found no negative effects after 1 year. Analysis of tissue from 13 participants who died due to ALS found that the transplanted cells had persisted and had made GDNF.
A 2023 analysis of two previous phase 1 clinical trials looked at the long-term effects of transplanting MSCs into the lumbar spinal cord of 19 people with ALS. At the time of analysis, four of the individuals were still living.
Researchers found that the average observed survival of trial participants was 118.8 months — almost 10 years. This was longer than the predicted average survival of 70.79 months, a little under 6 years.
The Food and Drug Administration (FDA) has not approved a stem cell treatment for ALS. People receiving stem cell treatments for their ALS receive them through participation in a clinical trial.
NurOwn, developed by BrainStorm Cell Therapeutics, uses MSCs to boost levels of factors that support the health and survival of nerve cells.
If you’ve been diagnosed with ALS, your care team may suggest that you participate in a clinical trial.
Clinical trials test the safety and effectiveness of new or updated treatments before they’re made more widely available.
You can find a list of clinical trials for ALS that are currently recruiting here.
Your care team is also a valuable resource. They can help to give you an idea of current or upcoming clinical trials that you may be eligible for.
The current treatment options for ALS include:
- medications like riluzole (Rilutek, Tiglutik, Exservan) or edaravone (Radicava), which can help to lower damage to motor neurons and slow declines in daily functioning, respectively
- other medications to help manage symptoms like muscle cramping, sleep trouble, or anxiety and depression
- physical and occupational therapy to aid in boosting strength, flexibility, and independence
- speech therapy or devices to help you communicate
- psychotherapy to help you mentally and emotionally cope with the effects of ALS
- respiratory support, including noninvasive ventilation or mechanical ventilation
ALS is progressive. That means that it continues to worsen as time passes.
The effects of ALS eventually lead to paralysis, which can also affect the muscles that help you to breathe. As such, most people with ALS die due to respiratory failure.
Can a bone marrow transplant cure ALS?
No. However, researchers are investigating bone marrow transplants to treat ALS. This research is in its early stages.
One 2022 clinical trial showed no difference in ALS progression after a bone marrow transplant. Another
What other conditions are stem cells being researched for?
In addition to ALS, stem cell therapies are being investigated for a wide variety of conditions. A few examples include:
- other neurodegenerative diseases like Parkinson’s disease and Alzheimer’s disease
- spinal cord injuries
- heart disease
- type 1 diabetes
What are FDA-approved stem cell treatments?
Hemopoietic stem cell products, which contain stem cells that can differentiate into various blood cells, are currently the
Stem cells are an attractive potential treatment for ALS. It’s hoped that stem cell therapy could slow or reverse the effects of ALS.
However, research into stem cells for ALS is still generally in its early stages, and no stem cell-based ALS treatments have been approved by the FDA. Overall, more research into the safety and effectiveness of these treatments is needed.
People with ALS who receive stem cell therapy do so through clinical trials. If you have ALS and are interested in participating in a clinical trial, talk with your care team. They can help you find clinical trials that you could be eligible for.