By:
Louis A. Cona, MD
Reviewed:
Robert J. Hancock
Stay informed about the latest medical research on the use of stem cell therapy in treating ALS. Learn how this innovative treatment approach has the potential to slow the degeneration of motor neurons and improve the quality of life for patients with ALS.
Stem cell therapy for ALS
Mesenchymal stem cell therapy has already shown strong therapeutic potential in different clinical fields. In particular, mesenchymal stem cells (MSCs) may exert their action by differentiating toward a specific cell type or by releasing further 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, the loss of motor neurons may also 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)
A new ALS treatment option
Stem cell therapy for Amyotrophic Lateral Sclerosis is a treatment approach that involves using stem cells, undifferentiated cells that can develop into various specialized cells, to replace or repair damaged or lost cells in the brain associated with ALS. The stem cells are administered into the body via intravenous injection and will find areas of inflammation and damage via homing mechanisms.
The primary mechanisms of stem cells are to reduce inflammation (including neuroinflammation) and modulate the immune system. Stem cell therapy aims to prevent disease progression for an extended period through immune modulation. A secondary benefit of stem cell treatment for ALS is to help prevent further loss of neurons to slow progression and improve motor symptoms, tremors, stiffness, and difficulty with movement.
How do stem cells help ALS?
Stem cells adopt a supportive role by providing a nurturing and neuroprotective microenvironment that improves dangerous conditions for diseased motor neurons, thereby slowing neurodegeneration and neuronal death. Transplanted stem cells, in this capacity, secrete neurotrophic factors and differentiate into non-diseased, non-neuronal cells, such as astrocytes and microglia, or 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.
Despite numerous clinical trials, there is no cure for ALS, aka Lou Gehrig's Disease; current therapies are palliative and only extend survival by a few months. Stem cell therapy is attractive for ALS because it addresses complex disease development through multiple mechanisms.
The premise of stem cell therapy for Amyotrophic Lateral Sclerosis (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 the degeneration of motor neurons. (4)
ALS stem cell treatment
Stem cell therapy may be a viable treatment option for ALS. Stem cells may be feasible for sustaining and nurturing 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.
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 dramatically benefits physicians worldwide seeking different forms of treatment, all without causing further harm to ALS patients.
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 protect a patient's healthy neurons and 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 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.
What is ALS?
Amyotrophic Lateral Sclerosis, often called Lou Gehrig's disease, is tricky to understand and treat. It affects approximately one to two per 100,000 people in the United States annually. 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.
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 and muscle weakening over time. This automatic spasticity results in worsening symptoms.
Types of Amyotrophic Lateral Sclerosis
Generally, ALS is categorized into two types: upper motor neuron disease and lower motor neuron disease. Upper motor neuron disease affects the nerves in the brain, while lower motor neuron disease affects the nerves from the spinal cord or brainstem.
In both cases, the motor neurons are damaged and ultimately die. As a result of the progressive degeneration of motor neurons, ALS is a fatal disease, with the average life expectancy after diagnosis being between two to five years. However, some patients may live for more extended periods.
ALS Cure: Does it exist?
At present, there is no cure for ALS. However, there are several treatment options, including stem cell therapy, Riluzole, occupational therapy, and gene therapy, which may help manage symptoms and slow the progression of the disease, as well as ongoing research for potential cures and treatments.
Medications: Riluzole is the only FDA-approved medication for ALS, and it can help slow the progression of the disease. Other drugs, such as edaravone, can also help manage symptoms such as muscle cramps and spasms.
Physical therapy: Physical therapy can help maintain muscle strength and function, as well as help with mobility, balance, and coordination.
Occupational therapy: Occupational therapy can help with activities of daily living, such as eating, bathing, and dressing.
Speech therapy: Speech therapy can help with communication, including using communication devices.
Nutritional support: Nutritional support can help with maintaining weight and preventing malnutrition.
Respiratory therapy: Respiratory therapy can help with breathing problems, a common complication of ALS.
Stem Cell Therapy: Stem cell therapy is a promising area of research for ALS. Studies have shown that stem cells can migrate to the site of injury, differentiate into the missing cells, and release various trophic factors that can promote the survival of the remaining cells. Some studies focus on using mesenchymal stem cells to reduce inflammation and promote the survival of motor neurons.
Gene Therapy: Gene therapy is another promising area of research for ALS. Some studies have focused on delivering specific genes that can help protect or repair motor neurons. For example, delivery of the neurotrophic factor GDNF has been shown to improve the survival of motor neurons in animal models of ALS. Another example is the delivery of a therapeutic gene, such as SOD1, that can help reduce the toxic effects of mutant SOD1, a protein known to cause familial ALS. However, more research is needed to fully understand the safety and efficacy of gene therapy in humans.
Small molecule therapy: Recent studies have focused on identifying small molecules that can promote the survival of motor neurons and reduce inflammation.
Immunomodulation therapy: Studies have shown that inflammation plays a vital role in the progression of ALS. Some recent studies have focused on the use of immunomodulation therapy to reduce inflammation and improve the survival of motor neurons. For example, some studies have shown that the drug, Rituximab, can reduce inflammation and improve the survival of motor neurons in animal models of ALS.
ALS Treatment
Amyotrophic Lateral Sclerosis treatments are a highly sought-after topic. Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease affecting the brain and spinal cord nerve cells. While there is currently no cure for ALS, several Amyotrophic Lateral Sclerosis treatments are available to help manage symptoms and improve the quality of life for those with the condition.
How is ALS treated?
According to recent peer-reviewed research, one promising treatment option is the use of Riluzole, a medication that has been shown to slow the progression of ALS and extend survival by a few months. Other potential treatments include physical therapy, speech therapy, stem cell therapy, and respiratory care to help manage symptoms such as muscle weakness, difficulty speaking, and difficulty breathing. Individuals with ALS must work with a medical team to determine the most appropriate treatment plan.
ALS Research using stem cells
Mesenchymal stem cells (MSCs) are a type of stem cell that can differentiate into various cell types, including cells of the musculoskeletal system. Some studies have suggested that MSCs may have potential as an Amyotrophic Lateral Sclerosis treatment, as they can modulate the immune system and reduce inflammation, which may be beneficial in managing neurodegenerative conditions such as ALS.
Several studies have explored using mesenchymal stem cells (MSCs) as a potential treatment for ALS. A systematic review published in the Journal of Neuroimmunology in 2016 analyzed the results of 13 clinical trials that used mesenchymal stem cells (MSCs) to treat ALS. The review found that MSCs were generally well-tolerated and had a favorable safety profile, with no serious adverse effects reported in the studies reviewed.
However, the review also found that the results of the clinical trials were mixed and that further research is needed to fully understand the potential of mesenchymal stem cells (MSCs) as a treatment for ALS. Some studies reported improvements in muscle strength and function, while others found no significant benefits.
Overall, using mesenchymal stem cells (MSCs) as a treatment for ALS is still a topic of ongoing research and debate. More studies are needed to understand this approach's potential benefits and risks. Individuals with ALS must work with their medical team to determine the most appropriate treatment plan.
What is Nurown stem cell treatment?
Nurown is a type of cellular therapy developed to treat ALS (Amyotrophic lateral sclerosis), also known as Lou Gehrig's disease. Nurown is a proprietary, autologous (self-donated) stem cell therapy comprising a patient's bone marrow-derived mesenchymal stem cells (MSCs) that are expanded in the lab and delivered back to the patient. The treatment is designed to slow the progression of ALS by promoting the survival and growth of motor neurons affected by the disease.
Nurown for ALS has been developed and manufactured by BrainStorm Cell Therapeutics. The therapy is still in the early stages of development and has undergone a Phase 2 clinical trial. The test results demonstrated a statistically significant improvement in the rate of decline in the primary endpoint of ALSFRS-R, a measure of ALS disease progression, as well as substantial improvements in secondary endpoints, including muscle strength and quality of life.
When will Nurown be available?
It's important to note that Nurown is not yet FDA-approved, and more research is needed to fully understand its safety and efficacy in treating ALS. Additionally, The therapy is not available for general use and only in clinical trials.
How is ALS diagnosed?
The diagnosis of Amyotrophic lateral sclerosis (ALS) is based on a combination of clinical examination, laboratory tests, and imaging studies. The process of diagnosis usually starts with a thorough medical history, physical examination, and neurological examination.
How to diagnose ALS
A neurologist usually provides an ALS diagnosis as they will have experience in neuromuscular disorders. The diagnosis can often take some time to confirm, as the symptoms and progression of the disease can vary significantly from person to person.
Once a diagnosis of ALS is made, the neurologist will work with the patient and their family to develop a treatment plan, including medications, physical therapy, occupational therapy, speech therapy, and other support services. It is important to note that the definitive diagnosis of ALS can only be confirmed by autopsy or by demonstrating a superoxide dismutase 1 (SOD1) gene mutation.
Medical history: The doctor will ask questions about the patient's symptoms, including when they started, how they have progressed, and what makes them better or worse.
Physical examination: The doctor will check for muscle weakness, atrophy, and spasticity. They may also check for reflexes, muscle tone, and sensation.
Neurological examination: The doctor will check for signs of muscle weakness, atrophy, and spasticity. They may also check for reflexes, muscle tone, and sensation.
Laboratory tests: The doctor may order blood tests to check for other conditions that can cause similar symptoms, such as thyroid disease or anemia.
Imaging studies: The doctor may order imaging studies, such as an MRI or CT scan, to rule out other conditions that can cause similar symptoms, such as a spinal cord tumor or multiple sclerosis.
Electromyography (EMG) and nerve conduction study (NCS) tests: These tests can help to confirm the diagnosis of ALS by measuring the electrical activity of the muscles and nerves.
Biopsy: A muscle or nerve biopsy can be performed to confirm the diagnosis of ALS, but it's not commonly done because it's invasive.
Genetic testing: Genetic testing can be used to identify genetic mutations that are associated with familial ALS.
It's important to note that no single test can confirm a diagnosis of ALS. The diagnosis is based on a combination of clinical findings, laboratory tests, and imaging studies, and it is vital to exclude other conditions that may have similar symptoms.
What are the symptoms of ALS
The symptoms of Amyotrophic lateral sclerosis (ALS) can vary depending on the individual and the progression of the disease. However, some common ALS symptoms include the following:
Weakness: This is usually the first symptom of ALS. It can start in the muscles of the hands, feet, arms, or legs and gradually spread to other body parts.
Muscle twitching or cramping: These symptoms can be caused by the degeneration of motor neurons.
Loss of muscle mass: As the motor neurons die, the muscles they control can shrink or atrophy.
Difficulty speaking: As ALS progresses, it can become harder to speak, swallow, or breathe.
Fatigue: People with ALS can experience increased fatigue, especially during increased activity.
Loss of coordination and balance: As the disease progresses, coordinating movements and maintaining balance can become challenging.
Changes in speech: As the disease progresses, it can become harder to speak, swallow, or breathe.
Difficulty swallowing: As ALS progresses, it can become difficult to swallow, leading to malnutrition and weight loss. Swallowing difficulties caused by ALS can result in malnutrition and weight loss due to insufficient protein and calorie intake. When this occurs, the body starts to break down muscle tissue for energy and protein, accelerating muscle weakness. A PEG (percutaneous endoscopic gastrostomy) tube or feeding tube can provide adequate nutrition and prevent further weight loss. However, there is also a risk that food or liquid may accidentally be inhaled into the airways instead of being directed to the stomach when using these tubes.
Breathing problems: As the disease progresses, breathing can become complicated, leading to respiratory failure.
It is important to note that not all people with ALS will experience all of these symptoms, and the progression of the disease can vary significantly from person to person. If you are experiencing any of these symptoms, speaking with a healthcare professional is essential.
Can drugs help ALS patients?
Currently, drugs that slightly increase the length of life and improve quality of life are available, but there is no accepted cure for ALS today. In recent years, there has been a push to raise awareness of ALS 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.
New drugs for ALS
Several new drugs are currently in development or have recently been approved for the treatment of Amyotrophic Lateral Sclerosis (ALS). Here is a list of some of the new drugs that are being explored for the treatment of ALS:
Radicava (Edaravone): This medication was approved by the US Food and Drug Administration (FDA) in 2017 for treating ALS. It is believed to work by reducing oxidative stress in the body, which may help slow the progression of the disease.
Tirasemtiv: This medication is in phase 3 clinical trials for treating ALS. It is believed to work by activating a protein called fast skeletal muscle troponin, which may help improve muscle function in people with ALS.
RG-2: This drug is in phase 2 clinical trials for treating ALS. It is believed to work by reducing inflammation and oxidative stress in the body, which may help slow the progression of the disease.
NurOwn: This experimental medication is being developed as a potential treatment for ALS and is currently in phase 3 clinical trials. It is a cell-based therapy that uses mesenchymal stem cells (MSCs) to help modulate the immune system and reduce inflammation in the body.
Vamorolone is in phase 2 clinical trials for treating ALS and other muscle-wasting conditions. It is believed to work by reducing inflammation and preserving muscle mass.
It is important to note that these drugs are still in the early stages of development and may not be available for widespread use for some time. It is also important to remember that the effectiveness and safety of these drugs will need to be demonstrated in clinical trials before they can be approved for use in the general population.
Can stem cell therapy cure ALS?
There is currently no cure for ALS, and treatment options are limited to medications that can slow the progression of the disease and supportive care to manage symptoms.
Several clinical trials have investigated stem cell therapy as a potential treatment for ALS. Some of these studies have shown promising results, while others have not.
One study published in the Journal of the Neurological Sciences in 2014 examined the use of mesenchymal stem cells (MSCs) in patients with ALS. MSCs are a type of adult stem cell that can differentiate into various kinds of cells, including muscle cells. The study found that mesenchymal stem cells (MSCs) improved muscle strength and function in patients with ALS, and the treatment was well-tolerated, with no serious adverse events reported.
Another study published in the journal Stem Cells Translational Medicine in 2014 examined the use of neural stem cells (NSCs) in patients with ALS. NSCs are a type of stem cell that can differentiate into nerve cells, including neurons and glial cells. The study found that NSC transplantation was associated with improved survival and function in patients with ALS, and the treatment was well-tolerated.
While these studies suggest that stem cell therapy may have potential as a treatment for ALS, more research is needed to understand the effectiveness and safety of this approach entirely. It is important to note that stem cell therapy is not a proven treatment for ALS and should be approached with caution. Suppose you are considering stem cell therapy as a treatment for ALS. In that case, consulting with a qualified healthcare provider and carefully weighing the potential risks and benefits is essential.
References:
(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: https://doi.org/10.1001/jamaneurol.2015.4321
(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 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6431432/.
(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 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6697143/
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