Stem Cell Therapy, An ALS Breakthrough in 2023?

Louis A. Cona, MD
Updated on
Dec 6, 2022

minute read

Stem cell therapy may have the ability to slow the progression of ALS.

Learn more about stem cell therapy and the science behind it.

Introduction to ALS

Amyotrophic Lateral Sclerosis, or ALS, is a relatively tricky disease to understand and treat. It affects approximately one to two per 100,000 people in the United States every year. 

Nearly 95% of ALS cases are caused by unknown factors, with only around 5% being genetically inherited from parents. Although ALS usually strikes around age 50-60, it can affect people of any age. 

Is there a cure for ALS?

Unfortunately, there is no known cure for Amyotrophic Lateral Sclerosis (ALS), and the current prognosis is two to four years from onset. Recent advances in stem cell technology have provided both new tools for researchers to fight ALS, as well as possible new treatments for patients themselves.  Stem cell therapy may be able to delay the progression of the disease state. However, more long term research studies should be conducted to establish treatment efficacy.

Stem cell therapy for ALS

Mesenchymal stem cell therapy has already shown a strong therapeutic potential in different clinical fields. In particular, mesenchymal stem cells (MSCs) may exert their action differentiating toward a specific cell type or through the releasing of different growth and trophic factors.

Clinical models have shown evidence that mesenchymal stem cells (MSCs) may represent a promising approach to treat ALS; MSC transplantation may delay the disease onset and progression and therefore increase lifespan. Furthermore, also the loss of motor neurons may be reduced, resulting in a delay in motor function loss. The results obtained from preclinical studies have encouraged the administration of mesenchymal stem cells (MSC) in ALS patients. (3)

Stem cells adopt a supportive role by providing a nurturing and neuroprotective microenvironment that improves detrimental conditions for diseased motor nueurons, thereby slowing neurodegeneration and neuronal death. 

Transplanted stem cells in this capacity secrete neurotrophic factors, differentiate into non-diseased, non-neuronal cells, such as astrocytes and microglia, or into modulatory neurons that synapse with diseased motor neurons (MN). Preclinical studies are encouraging and have demonstrated the potential applicability of stem cells to treat ALS. 

There is no cure for ALS despite numerous clinical trials; current therapies are palliative and only extend survival a few months.  This makes stem cell therapy is an attractive approach for ALS because it addresses the complex disease development through multiple mechanisms.

The premise of stem cell therapy for ALS is based on improving the diseased microenvironment. Transplanted stem cells secrete neurotrophic factors and differentiate into supportive cells, such as astrocytes and microglia, generating a neuroprotective milieu that can slow degeneration of motor neurons. (4)

What is ALS?

Amyotrophic Lateral Sclerosis (ALS) occurs when the body starts experiencing the death of neurons controlling voluntary muscles. 

ALS affects both upper and lower motor neurons, meaning patients with the disease begin to experience both involuntary spasticity as well as weakening of the muscles over time. This involuntary spasticity results in worsening symptoms.

Symptoms of ALS

  • Difficulty speaking
  • Difficulty moving
  • Difficulty breathing
  • Muscle cramps
  • Muscle stiffness
  • Slurred speech

Can drugs help ALS patients?

There are currently drugs available, which both slightly increase length of life, as well as improve quality of life, but there is no accepted cure for ALS today. There has been a push in recent years to raise awareness to ALS in an attempt to promote research into finding a cure. This increase in awareness was most notably seen in 2014 with the introduction of the ALS Ice Bucket Challenge.

Pictured: Group participating in the ALS Ice Bucket Challenge, a social movement to create awareness for Amyotrophic Lateral Sclerosis

ALS Stem cell treatment

Stem cell therapy may be a viable treatment option for ALS.  Stem cells may be a viable solution to sustain and nurture diseased motor neurons.  (1)

According to a recent study conducted by Goutman et al.

“The premise of stem cell therapy for ALS aims to improve the diseased microenvironment. While stem cells are unable to replace diseased motor neurons directly, transplanted stem cells secrete neurotrophic factors and differentiate into supportive cells, such as astrocytes and microglia, generating a neuroprotective milieu that can slow degeneration of motor neurons.” (1) 

Researchers have turned to stem cells in the fight against ALS for two main reasons.

  1.  Doctors need an ample supply of ALS sample cells on which to test treatments. Fortunately, a type of stem cell called an Induced Pluripotent Stem Cell (IPSC) can closely mimic the neurons affected by ALS. Researchers need only small skin samples from ALS patients to create an indefinite amount of IPSCs in the lab, allowing for continuous testing on genetically identical cells to the patients. This capability provides a massive benefit to physicians around the world seeking different forms of treatment, all without causing any further harm to ALS patients.
  2. Stem cells are being used in trials to treat ALS directly. Stem cells can seek out damage in the body and replace cells of any type. For this reason, stem cell transplants are currently used to both protect a patient’s healthy neurons, as well as potentially grow new cells to replace those that have died.

Stem cell trials for ALS published

A study conducted by Panayiota Petrou et al. in 2016, found stem cell therapy to be both safe and well-tolerated by ALS patients. 

“Among the 26 patients, 87% were defined as responders to either ALS Functional Rating Scale-Revised or forced vital capacity, having at least 25% improvement at six months after treatment in the slope of progression.” (2)

 Researchers hope treatments like this will be able to eventually slow to stop the progression of ALS, vastly improving the results of current drugs on the market.

Stem cell treatment for ALS at DVC Stem

Stem cell therapy may have the ability to slow the progression of ALS. This is conducted through stem cells ability to differentiate into unique types of supportive cells such as astrocytes and microglia (cells within the central nervous system). These supportive cells may have the ability to slow the degeneration of motor neurons within the CNS.

DVC Stem is a stem cell therapy pioneer, offering stem cell therapies for years and has become a cornerstone of the medical tourism industry. Located in the tropical paradise of Grand Cayman in the Western Caribbean, we offer patients a nearby alternative to travelling long distances and to less ideal locations. Our protocols are IRB approved, and our cells come from regulated, U.S. based, FDA compliant laboratories. We seek to offer the highest quality products, the latest available treatments for a variety of conditions, all combined with a world-class setting and service. Contact us today to request additional information.

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(1) Stephen A. Goutman, Masha G. Savelieff, Stacey A. Sakowski & Eva L. Feldman (2019) Stem cell treatments for amyotrophic lateral sclerosis: a critical overview of early phase trials, Expert Opinion on Investigational Drugs, 28:6, 525-543, DOI: 10.1080/13543784.2019.1627324

(2) Petrou P, Gothelf Y, Argov Z, et al. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016;73(3):337–344. doi:

(3) Gugliandolo, A., Bramanti, P., & Mazzon, E. (2019, March 10). Mesenchymal stem cells: A potential therapeutic approach for amyotrophic lateral sclerosis? Stem cells international. Retrieved December 8, 2021, from

(4) Goutman, S. A., Savelieff, M. G., Sakowski, S. A., & Feldman, E. L. (2019, June). Stem cell treatments for amyotrophic lateral sclerosis: A critical overview of early phase trials. Expert opinion on investigational drugs. Retrieved December 30, 2021, from

Note: This post is intended to provide general information about regenerative medicine, and related areas. All content provided in this blog, website, or any linked materials, including text, graphics, images, patient outcomes, and information, are not intended and should not be considered or used as a substitute for medical advice, diagnosis, or treatment. Please always consult with a professional and certified healthcare provider to discuss if a treatment is right for you.

About the author

Louis A. Cona, MD

Medical Director | DVC Stem

Dr. Cona has been performing stem cell therapy for over 12 years. He is a member of the World Academy of Anti-Aging Medicine (WAAAM). He is also a recognized member of the British Medical Association, the General Medical Council (UK), the Caribbean College of Family Physicians, and the American Academy of Family Physicians. He is the Medical Director for DVC Stem a world-renowned stem cell therapy clinic located in Grand Cayman.

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