The Food and Drug Administration (FDA) has not approved stem cell therapy for amyotrophic lateral sclerosis (ALS). However, people could participate in clinical trials involving this ALS treatment.

ALS is a progressive disease that affects nerve cells that control voluntary muscle movement and breathing. It causes the nerve cells to degenerate and die. As this occurs, the nerve cells stop sending messages to muscles, causing them to become weak.

Medical professionals formerly referred to ALS as Lou Gehrig’s disease.

This article outlines how stem cell therapy may work to treat ALS and discusses clinical trials and other treatments.

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Stem cells in the body can develop into any other type of cell. They also have the ability to self-renew.

During stem cell therapy, medical professionals replace damaged or dead cells with stem cells. These cells then carry out the function of the cells they replaced.

Stem cell therapy for ALS may offer different benefits.

According to the ALS Association, medical professionals may administer stem cells, which can deliver growth factors or protective molecules to nerve cells. This can help improve the health of the nerve cells and prevent them from dying.

Stem cell therapy may also use stem cells to replace damaged nerve cells. However, this may be challenging because the stem cells will need to mimic the appropriate connections between the surrounding neurons and muscles.

Types of stem cells for treating ALS

There are a number of different types of stem cells. A 2023 review stated that clinical and preclinical trials had taken place using the following stem cell types for treating ALS:

  • Mesenchymal stem cells (MSCs): These are stem cells that are present in:
  • Hematopoietic stem cells (HSCs): HSCs occur within the bone marrow and umbilical cord blood.
  • Neural stem cells (NSCs): NCSs occur within the brain of a fetus or in the subventricular zone of the adult brain.
  • Induced pluripotent stem cells (iPSCs): iPSCs come from adult somatic cells, which are any cells in the body that are not reproductive cells.

The FDA has not currently approved stem cell therapy for ALS. This means that a person can only make use of this treatment if they take part in a clinical trial.

Preclinical trials take place before human clinical trials occur. These trials aim to determine how safe a drug is. Clinical trials involve one or more human subjects and aim to evaluate the effects of certain medical interventions.

A number of preclinical and clinical trials have taken place to evaluate stem cell therapy in relation to ALS.

A 2021 clinical trial aimed to evaluate the safety and efficacy of MSC therapy in 20 people with ALS. These individuals received injections of MSC at intervals of 3 to 6 months.

The trial concluded that MSC therapy was safe and reported no adverse events during treatment. The study authors also added that the treatment helped improve the rate at which ALS progressed in 13 of the 20 participants. However, further large-scale research is necessary.

A 2021 systematic review identified 748 studies on the safety and efficacy of stem cell therapy for ALS. However, only six of these studies underwent evaluation in the review.

The review stated that some studies showed that stem cell therapy had a positive effect on people with ALS. However, the authors added that other studies found no significant difference between stem cell treatment and the control group.

The authors concluded that more clinical trials are necessary to determine the benefit of stem cell therapy for those with ALS.

A person needs to speak with a doctor if they are interested in taking part in a clinical trial.

There is currently no cure for ALS. The current treatment options aim to slow the progression of the disease and improve a person’s quality of life.

Current treatments for ALS with FDA approval include:

  • Riluzole: This medication helps reduce damage to motor neurons. It does this by reducing levels of glutamate, which helps transport messages between nerve cells and motor neurons. Clinical trials suggest that riluzole may help prolong survival by a few months for people with ALS.
  • Edaravone: This medication is an antioxidant, which may help slow functional decline.
  • Tofersen: This medication may help decrease one of the markers of damage to neurons. More studies are necessary to determine if this is the case. The FDA has only approved it for ALS relating to a mutation in the SOD1 gene.

The FDA approved the use of sodium phenylbutyrate/taurursodiol (Relyvrio) in 2022. This medication was proposed to block stress signals in cells, which may help prevent nerve cell death.

However, a large clinical trial failed to confirm findings from previous trials, so the manufacturer removed the drug from the market in 2024.

Stem cell therapy for amyotrophic lateral sclerosis (ALS) does not have approval from the Food and Drug Administration (FDA). People need to speak with a doctor if they are interested in taking part in a clinical trial.

When treating ALS in a stem cell therapy clinical trial, healthcare professionals may inject a person with stem cells that can deliver growth factors or protective molecules to damaged nerve cells. This may help improve the health of these cells and prevent them from dying.

Stem cell therapy may also use stem cells to replace nerve cells that ALS has damaged.