ALS Stem Cell Therapy: Can Cell-Based Treatments Help Amyotrophic Lateral Sclerosis?

Stem cell therapy offers new hope for ALS by aiming to slow disease progression, protect motor neurons, and improve quality of life where current treatments remain limited.

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that damages motor neurons, leading to muscle weakness, speech difficulties, and loss of independence.

Current treatments like riluzole and edaravone offer only modest benefits, and most patients with amyotrophic lateral sclerosis live three to five years after diagnosis. This makes the search for new therapies urgent, with stem cell treatment emerging as a promising option to slow progression and improve quality of life.

What Is ALS?

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease. It damages motor neurons, the nerve cells that carry signals from the brain and spinal cord to muscles. As these cells die, muscles weaken and gradually lose the ability to function.

ALS typically begins with symptoms such as muscle twitching, stiffness, or weakness in the arms or legs. Over time, patients may develop difficulties with speech, swallowing, and breathing. The disease usually advances steadily, leading to increasing disability.

ALS does not affect thinking ability in most patients, though some may develop cognitive or behavioral changes. Because the disease targets voluntary muscle control, everyday tasks become more difficult as it progresses.

What Are Current Treatments for ALS?

Treatments for ALS focus on slowing disease progression, easing symptoms, and maintaining quality of life. At present, there is no cure.

Two drugs, riluzole and edaravone, are approved worldwide as disease-modifying therapies. Riluzole helps extend survival by reducing damage from excess glutamate, a neurotransmitter that harms nerve cells when present in high amounts. Edaravone acts as an antioxidant, lowering oxidative stress that contributes to neuron loss.

Supportive care plays a central role. Non-invasive ventilation improves breathing comfort and can extend survival. Nutritional support, including feeding tubes when necessary, helps patients maintain weight and strength. Physical, occupational, and speech therapy all contribute to preserving independence and communication.

Beyond these established options, researchers are testing new medications, gene therapies, and cell-based approaches. These aim to target the disease earlier and more precisely.

Which therapies for ALS are approved today?

Several therapies are officially approved for ALS, though none cure the disease. They focus on slowing progression or easing symptoms.

• Riluzole: The first widely approved ALS drug. It reduces glutamate-related nerve cell damage and modestly extends survival.
• Edaravone: An intravenous antioxidant therapy. It lowers oxidative stress in neurons and may slow functional decline in certain patients.
• AMX0035 (sodium phenylbutyrate/taurursodiol): Approved in the US and Canada. It targets cellular stress pathways and has shown benefits in slowing disease progression.
• Tofersen: An antisense oligonucleotide approved for patients with SOD1 gene mutations. It reduces production of the toxic protein that drives motor neuron death in this subgroup.
• NeuroNata-R (lenzumestrocel): The first licensed stem cell therapy for ALS, approved in South Korea in 2014. It uses autologous mesenchymal stem cells from bone marrow to provide neuroprotection, immune modulation, and anti-inflammatory effects. A phase 3 trial is planned for US and European markets.

Together, these therapies represent meaningful progress but highlight how limited treatment options remain. Most ALS patients still rely on a combination of drug therapy and multidisciplinary supportive care.

What are the limits of existing ALS treatment options?

Approved therapies for ALS offer only partial benefit. They can slow disease progression but cannot stop or reverse it. Most patients continue to lose muscle function despite treatment.

• Modest survival gains: Riluzole extends life by a few months on average. Edaravone and AMX0035 slow decline but their effect is measurable only in subsets of patients.
• Restricted eligibility: Some therapies, like edaravone, show effectiveness mainly in early-stage disease. Tofersen applies only to patients with a rare SOD1 mutation.
• Symptom relief but not cure: Supportive measures—non-invasive ventilation, feeding support, and rehabilitation—prolong survival and improve quality of life but do not alter disease biology.
• Regional access limits: NeuroNata-R, the only approved stem cell therapy, is available only in South Korea. Other regions are awaiting results from late-stage trials.

These limitations highlight the need for new approaches, including broader genetic therapies, more effective disease-modifying drugs, and accessible cell-based treatments.

How do new treatments for ALS aim to improve outcomes?

New ALS treatments are designed to go beyond slowing progression of disease for the patients with ALS. They target the biological mechanisms that drive motor neuron death, aiming to preserve function for longer.

• Gene-targeted therapies: Drugs like tofersen use antisense oligonucleotides to silence harmful mutations, addressing the root cause in select patients. Similar approaches are being tested for other ALS-related genes.
• Cell-based therapies: Experimental stem cell treatments, including mesenchymal stem cell therapy, seek to repair or protect motor neurons by releasing growth factors, reducing inflammation, and supporting the neural environment.
• Neuroprotective agents: New compounds are being studied to shield motor neurons from oxidative stress, mitochondrial failure, and toxic protein build-up.
• Combination therapies: Trials are exploring whether combining drugs, or pairing drugs with cell therapy, can provide greater and more sustained benefits.

The shared goal of these emerging treatments is to extend survival while preserving independence and quality of life, something current therapies only partially achieve.

What Role Do Stem Cells Play in ALS Research?

Stem cells are central to ALS research because they offer both therapeutic and investigative potential. Unlike drugs that target single pathways, stem cells act on multiple disease mechanisms.

• Neuroprotection: Stem cells can release growth factors, such as brain-derived neurotrophic factor, which support motor neuron survival.
• Immunomodulation: They influence immune activity by reducing harmful inflammation around neurons. Mesenchymal stem cells, for example, shift microglia toward a more protective state.
• Repair and regeneration: Although current therapies cannot replace lost motor neurons, stem cells may help repair damaged neural environments, improving conditions for surviving neurons.
• Disease modeling: Induced pluripotent stem cells (iPSCs) created from ALS patient samples allow scientists to study disease processes in the lab and test new therapies more precisely.

Research in this area has already led to experimental treatments, including NeuroNata-R in South Korea, and informs the design of clinical trials worldwide.

Why are stem cell therapies studied for ALS?

Stem cell therapies are studied in ALS because they address multiple disease pathways that drugs alone cannot. ALS is driven by complex processes like motor neuron loss, inflammation, oxidative stress, and toxic protein build-up, making single-target therapies less effective.

Researchers focus on stem cells because they:

• Provide protective support: Stem cells release growth factors that keep motor neurons alive longer.
• Modulate harmful inflammation: They can rebalance the immune system, shifting microglia and T cells toward protective roles.
• Stabilize the neural environment: By improving conditions around remaining neurons, stem cells help preserve muscle control.
• Offer personalized approaches: Autologous stem cell treatments use a patient’s own cells, lowering rejection risk.

Because stem cells can influence several mechanisms at once, they are viewed as a promising option to slow ALS progression where current treatments fall short.

How do different types of stem cells work in ALS research?

Different stem cell types are being investigated to treat ALS because each has unique properties that may protect or support motor neurons.

• Mesenchymal stem cells (MSCs): Usually derived from bone marrow or fat tissue. They release growth factors, reduce inflammation, and promote repair in the neural environment. MSC-based therapy, such as NeuroNata-R in South Korea, has shown the ability to slow ALS progression in early trials.
• Neural progenitor cells: These are more specialized cells that can become neurons or glial cells. In ALS research, they are studied for their potential to replace damaged support cells and enhance motor neuron survival.
• Induced pluripotent stem cells (iPSCs): Created by reprogramming adult cells back to a stem-like state. They are valuable both for modeling ALS in the lab and for exploring patient-specific therapies. iPSCs help researchers test new drugs and study how ALS develops in different genetic backgrounds.
• Embryonic stem cells (ESCs): Pluripotent cells capable of becoming any cell type. Their use is more limited due to ethical considerations but they remain important in basic ALS research.

Each type contributes differently: MSCs are furthest in clinical testing, while iPSCs and neural progenitors expand the understanding of ALS biology and future treatment possibilities.

What are mesenchymal stem cells and how might they help ALS patients?

Mesenchymal stem cells (MSCs) are adult stem cells most often taken from bone marrow, adipose tissue, or umbilical cord blood. They can differentiate into bone, cartilage, and fat cells, but their value in a motor neuron disease like ALS lies in their ability to support and protect motor neurons rather than replace them.

For ALS patients, MSCs may help in several ways:

• Neuroprotection: They secrete growth factors, including brain-derived neurotrophic factor, which improve motor neuron survival.
• Immune modulation: MSCs influence the immune response, shifting it from a damaging inflammatory state to a more protective balance.
• Anti-inflammatory action: They help convert microglia, the brain’s immune cells, from an aggressive form to a supportive one.
• Stabilizing the neural environment: By improving the surrounding conditions, MSCs reduce stress on surviving motor neurons and slow disease progression.

Clinical studies, including those on NeuroNata-R in South Korea, suggest MSC therapy can slow functional decline in ALS patients. While not curative, MSCs represent the most advanced form of stem cell therapy currently being tested for ALS.

What is the role of induced pluripotent stem cells in ALS research?

Induced pluripotent stem cells (iPSCs) are created by reprogramming adult cells, such as skin or blood cells, back into a stem-like state. They can then be directed to form many cell types, including neurons and glial cells. In ALS research, iPSCs are valuable for both laboratory modeling and therapeutic exploration.

Their main roles include:

• Modeling ALS in the lab: iPSCs derived from ALS patients carry the same genetic mutations and disease traits. Scientists use them to study how motor neurons degenerate over time.
• Testing therapies: Patient-specific iPSC models allow researchers to evaluate new drugs or gene therapies in human-derived cells before moving to clinical trials.
• Personalized medicine: Because iPSCs can be created from an individual patient, they open the possibility of designing tailored therapies that reflect each person’s disease profile.
• Exploring cell replacement: While not yet ready for clinical use, iPSC-derived neurons and glial cells may eventually serve as a source for transplantation to restore lost or damaged cells.

Although still experimental, iPSCs give researchers a powerful tool to understand ALS biology and accelerate discovery of new treatment strategies.

What Is Stem Cell Therapy for ALS?

Stem cell therapy for ALS is an experimental approach that uses stem cells to protect, repair, or support the motor neurons damaged by the disease. Unlike conventional drugs, which target specific pathways, stem cells can influence several processes at once.

The goal is not to cure ALS but to slow its progression and preserve muscle function for longer. Stem cells may:

• Release growth factors that help motor neurons survive.
• Reduce inflammation and shift immune responses toward a more protective state.
• Improve the surrounding environment of nerve cells, making it less toxic and more stable.
• Potentially replace or support lost glial cells that normally protect neurons.

Different types of stem cells are under study, including mesenchymal stem cells, neural progenitor cells, and induced pluripotent stem cells. Clinical trials are testing whether these approaches can safely delay disability and improve quality of life.

How does stem cell therapy for ALS work?

Stem cell therapy for ALS works by delivering cells that can protect and support motor neurons rather than directly replacing them. The approach targets several disease mechanisms at once, which is important in a condition as complex as ALS.

Key ways stem cells act include:

• Neurotrophic support: They release growth factors such as brain-derived neurotrophic factor, which strengthen and prolong motor neuron survival.
• Immune regulation: Stem cells help rebalance the immune system, reducing harmful inflammation around neurons.
• Anti-inflammatory effects: They shift microglia and other immune cells into a state that protects rather than damages nerve tissue.
• Neural environment repair: By stabilizing the space around neurons, stem cells improve communication between nerve and muscle, slowing muscle weakness.

In most experimental protocols, stem cells are injected into the spinal fluid (intrathecal) or directly into nervous tissue. From there, they act as supportive “biological factories” that release protective molecules and reshape the disease environment.

How Stem-cell Therapy Performed for ALS Treatment?

The process depends on the type of stem cells being used, but most approaches follow a similar pattern.

• Cell collection: For autologous therapies, stem cells are harvested from the patient’s own body, often from bone marrow or fat tissue. In some studies, donor-derived or laboratory-prepared cells are used.
• Cell preparation: The collected cells are isolated, expanded, and sometimes modified in specialized laboratories. This ensures enough cells are available and that they meet safety and quality standards.
• Delivery method: Most clinical trials use intrathecal injections, where cells are introduced into the cerebrospinal fluid. This allows them to reach the spinal cord and brain, the main sites of motor neuron damage. Other trials test direct injections into spinal cord tissue.
• Treatment course: In some protocols, patients receive more than one injection, spaced weeks or months apart, to maintain the effect. Additional monitoring ensures safety and tracks changes in function.

The procedure does not replace motor neurons directly. Instead, the transplanted stem cells act by releasing protective molecules, reducing inflammation, and supporting the neurons that remain.

How is cell-based therapy different from other ALS treatments?

Cell-based therapy differs from standard ALS treatments because it is designed to act on several disease processes at once, rather than targeting a single pathway.

• Mechanism of action: Approved drugs like riluzole and edaravone work chemically, reducing glutamate toxicity or oxidative stress. In contrast, stem cells function as living “biological factories,” releasing growth factors, calming inflammation, and stabilizing the environment around motor neurons.
• Personalization: Autologous therapies use a patient’s own stem cells, which lowers the risk of rejection. Most existing ALS drugs are the same for all patients, regardless of disease subtype.
• Potential for repair: While current treatments slow decline, stem cells may promote repair of the neural environment and support remaining neurons more directly.
• Broader impact: Conventional drugs typically provide modest survival benefits of a few months. Cell-based therapy is being studied for its potential to produce stronger and longer-lasting effects, though results are still preliminary.

This difference positions stem cell therapy as a complementary approach rather than a replacement for current treatments, with the aim of extending both survival and quality of life.

What Do Clinical Trials Show About Stem Cell Therapy for ALS?

According to a clinical trial conducted at Hanyang University in South Korea, repeated intrathecal injections of autologous bone marrow–derived mesenchymal stem cells slowed ALS progression for at least six months compared with controls, as measured by ALS Functional Rating Scale-Revised scores.

A study published in Stem Cells Translational Medicine confirmed the safety and feasibility of two intrathecal injections in seven ALS patients, showing no major adverse effects over twelve months.

Further evidence from a 2018 randomized controlled trial in Annals of Neurology demonstrated that NeuroNata-R, an MSC-based therapy, significantly reduced the rate of functional decline at four and six months compared with placebo.

Together, these findings suggest that stem cell therapy for ALS is safe in the short term and may slow disease progression. However, long-term benefits and survival impact remain unproven, and large phase 3 studies are ongoing to establish efficacy.

What results have recent clinical trials in ALS stem cell therapy reported?

Recent trials report acceptable short-term safety and mixed efficacy signals. Early studies using mesenchymal stem cells (MSCs) show slowed functional decline over months in some cohorts.

Lenzumestrocel (Neuronata-R, autologous BM-MSCs): A randomized controlled study reported a slower ALSFRS-R decline at 4 and 6 months versus controls after repeated intrathecal dosing, with good tolerability. Phase 1 work established feasibility and safety of two intrathecal injections over 12 months. Real-world, propensity-matched analyses suggest an association with longer survival.

Neural progenitor cell approaches: A 2022 study of engineered neural progenitors delivering GDNF showed target engagement and procedure safety with durable graft presence; clinical benefit signals require larger trials.

What Are the Benefits of Stem Cell Treatment for ALS Patients?

Stem cell treatment is studied in ALS because it can influence several disease processes at once. Unlike standard drugs, which target single pathways, stem cells act as living systems that provide broad support.

• Functional slowing: According to a 2018 randomized controlled trial published in Annals of Neurology, repeated intrathecal injections of bone marrow–derived mesenchymal stem cells (NeuroNata-R) slowed ALS Functional Rating Scale-Revised decline at four and six months compared with controls.
• Safety and feasibility: A 2015 study in Stem Cells Translational Medicine found that two intrathecal doses of autologous mesenchymal stem cells were well tolerated in seven ALS patients over 12 months.
• Potential survival impact: A 2022 real-world, propensity-matched analysis suggested that lenzumestrocel treatment was associated with longer survival compared to standard care.
• Neuroprotection and immune effects: Research indicates that transplanted cells release growth factors, modulate immune activity, and reduce inflammation, all of which help create a healthier environment for surviving motor neurons.

These benefits remain modest and time-limited in current data, but they suggest stem cell therapy may offer functional preservation and quality-of-life improvements for ALS patients.

What risks and limitations are associated with stem cell treatment for amyotrophic lateral sclerosis?

Stem cell therapy for ALS is still experimental. While most studies show that the procedure is feasible and generally safe, important risks and limits remain.

• Procedure-related risks: Intrathecal injections can cause headaches, back pain, or infections. Spinal cord–targeted delivery carries risks of nerve injury or inflammation.
• Uncertain long-term safety: Most published trials have followed patients for months to a few years. The long-term behavior of transplanted cells, including risks of abnormal growth or tumor formation, is not fully understood.
• Limited and short-term benefit: Evidence shows slowed functional decline for a few months in some patients, but no clear or lasting survival extension across all studies.
• Heterogeneous response: Not all patients benefit equally. Disease stage, genetic background, and immune profile may influence outcomes, but predictors of response remain unclear.
• Regulatory and cost barriers: Large, phase 3 trials are ongoing, but until results are available, access outside research settings is constrained. Treatment is resource-intensive and expensive.

Frequently Asked Questions

Can stem cells treat ALS?

Stem cells cannot cure ALS, but early studies suggest they may slow progression in some patients. Mesenchymal stem cells release growth factors, reduce inflammation, and support motor neurons. Benefits appear modest and time-limited, and large phase 3 trials are still underway to confirm safety, effectiveness, and long-term outcomes.

Where to get stem cell therapy for ALS?

Stem cell therapy for ALS is available only in limited settings. South Korea has approved NeuroNata-R for clinical use. In most countries, access is restricted to regulated clinical trials. Stem cell treatment is also available with GenCell, where therapies are delivered under medical supervision with a focus on safety and care.

How much is stem cell therapy for ALS?

The cost of stem cell therapy for ALS varies widely. In approved settings like South Korea, treatment with NeuroNata-R may cost tens of thousands of dollars. In private clinics, prices often range from $20,000 to $50,000 or more.

Stem cell therapy protocols are determined based on the patient’s age, weight, and disease progression or current condition. The final price is confirmed once the treatment protocol is defined.

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