Medical Review: Dr. Gerald Mastaw, MD – Board-Certified Physician
Last Updated: October 2025
What Is ALS?
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative condition that affects nerve cells in the brain and spinal cord. These motor neurons control voluntary muscles involved in movement, speech, and breathing.
As these neurons degenerate and die, communication between the brain and muscles is lost, leading to muscle weakness, stiffness, and gradual loss of mobility.
Common Symptoms
- Muscle weakness or stiffness
- Difficulty speaking or swallowing
- Twitching or cramping in arms or legs
- Breathing difficulty in advanced stages
Although ALS typically spares cognitive functions, it profoundly impacts physical independence and emotional well-being.
How ALS Is Treated Today
Currently, there is no cure for ALS. Most treatment options aim to slow disease progression, manage symptoms, and preserve function as long as possible.
Common Treatment Approaches
- Medications: such as Riluzole and Edaravone to reduce oxidative stress and slow neuron damage
- Rehabilitation: physical, occupational, and speech therapy to maintain strength and communication
- Respiratory support: non-invasive ventilation as breathing muscles weaken
- Nutritional care: feeding support and high-calorie diets to prevent weight loss
While these interventions improve comfort and quality of life, they do not halt the underlying neurodegeneration.
Exploring Regenerative Medicine for ALS
Regenerative medicine, including stem cell research, is an emerging field exploring ways to protect and support nerve cells affected by ALS.
Potential Roles of Stem Cells in ALS Research
- Supporting motor neuron survival
- Reducing inflammation within the central nervous system
- Modulating immune activity to create a healthier environment for surviving neurons
- Delivering growth factors that may promote cellular repair
Important:
Stem cell therapy for ALS remains experimental and is not FDA-approved.
Clinical trials are ongoing to evaluate safety, optimal dosing, and long-term outcomes.
Recent Clinical Studies in ALS and Regenerative Medicine
2025 – Regulatory T-Cells and Immune Support
Title: Treatment with Tregs Safe, May Slow ALS Progression: Trial
Institution: Columbia University – Read Study
Summary:
In this small Phase I study, six ALS patients received infusions of regulatory T-cells (Tregs) derived from donated umbilical cord blood.
The therapy (CK0803) was well-tolerated and appeared to slow ALS progression in several participants. Researchers noted improved inflammatory balance and encouraged larger controlled studies to confirm potential benefits.
2024 – Muse Cells in Phase II Safety Trial (Japan)
Title: Stem Cell-Based Therapy Deemed Safe in Phase 2 Study
Source: ALS News Today – Read Summary
Summary:
Five patients received injections of Muse cells, a unique, stress-tolerant type of mesenchymal stem cell.
Treatment was safe and well tolerated, with no serious adverse events. Some patients showed modest improvements in function, though not statistically significant due to small sample size. Investigators concluded the results justified a larger, double-blind study.
2023 – Neural Progenitor Stem Cell / GDNF Gene Therapy
Title: Regenerative Medicine: A New Path for ALS Treatment
Institution: Cedars-Sinai / Nature Medicine – Read Summary
Summary:
Eighteen ALS patients received spinal implants of engineered neural progenitor stem cells releasing glial cell line-derived neurotrophic factor (GDNF).
The combined cell-gene therapy met safety goals, with long-term cell survival and no serious side effects. Although the trial focused on safety, researchers described the outcome as “encouraging enough to proceed” to next-phase studies evaluating efficacy.
2020 – Wharton’s Jelly MSCs (Poland Study)
Title: Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: An Original Study
Journal: Stem Cell Reviews and Reports – PubMed
Summary:
Sixty-seven ALS patients received three spinal (intrathecal) infusions of Wharton’s Jelly-derived MSCs.
Results showed doubled median survival time compared to untreated controls. Approximately one-third of treated patients had slower disease progression, and about half remained stable.
The therapy was well-tolerated, and repeated infusions appeared beneficial for responsive patients. Larger controlled studies are recommended.
2019 – Neural Stem Cell Safety and Feasibility (Italy)
Title: Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in ALS
Journal: Cell Transplantation – PubMed
Summary:
Eighteen ALS patients underwent spinal cord injections of human neural stem cells.
Over five years of follow-up, no serious therapy-related complications were reported. Some participants experienced temporary slowing of disease progression during the first few months’ post-treatment.
This trial provided proof of feasibility for targeted spinal delivery and informed later-phase research with higher doses.
Is Stem Cell Therapy Right for You?
If you or a loved one is living with ALS, it’s understandable to explore innovative or complementary options alongside standard care.
Before pursuing any regenerative therapy:
- Discuss your case with a board-certified neurologist or regenerative medicine specialist
- Review available clinical trial data and FDA guidance
- Understand the experimental nature of stem cell-based approaches
- Set realistic expectations about potential outcomes and limitations
At Stemedix, we emphasize safety, scientific transparency, and patient education. Our consultations focus on helping individuals understand ongoing ALS research and whether participation in regenerative programs aligns with their personal goals and medical profile.
Medical Disclaimer
This page is for educational purposes only and does not constitute medical advice.
Stem cell or exosome-based treatments for ALS are not FDA-approved.
Individual results may vary. Always consult your physician or neurologist before pursuing any therapy.
References
- Columbia University. Treatment with Tregs Safe, May Slow ALS Progression. ALS News Today, 2025. Link
- Japan ALS Consortium. Stem Cell-Based Therapy Deemed Safe in Phase 2 Study. ALS News Today, 2024. Link
- Cedars-Sinai. Regenerative Medicine: A New Path for ALS Treatment. Nature Medicine, 2023. Link
- Sierakowski A. et al. Umbilical Cord MSCs in Amyotrophic Lateral Sclerosis. Stem Cell Rev Rep, 2020. PubMed
- Mazzini L. et al. Phase I Trial with Intraspinal Neural Stem Cells in ALS. Cell Transplantation, 2019. PubMed
If you’re interested in learning more about stem cell therapy for ALS, contact us for a personalized consultation. We’re here to help you explore your options with clarity and compassion.
Search our blog page to learn more: https://stemedix.com/blog/
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