Phase II Trial Shows Promise of Repeated Stem Cell Injections for ALS Treatment

Phase II Trial Shows Promise of Repeated Stem Cell Injections for ALS Treatment

Amyotrophic Lateral Sclerosis (ALS) is a degenerative disease that affects motor neurons in the brain and spinal cord, leading to muscle paralysis and death, typically within 3-5 years of onset. Despite two FDA-approved therapies, Riluzole, and Edravarone, which offer limited benefits, there remains no cure for ALS. 

Considering this, researchers have turned to Mesenchymal Stem Cells (MSCs), which have shown promise in animal models and preliminary human trials for neurodegenerative diseases, including ALS.

Understanding ALS and MSC Therapy

ALS is characterized by the rapid degeneration of motor neurons, leading to muscle paralysis. The exact cause of ALS is complex and not fully understood. About 10% of cases are familial, while 90% are sporadic. Existing treatments only modestly slow disease progression and extend survival by a few months.

Stem cells, particularly MSCs, have shown potential in neuroprotection and immunomodulation. MSCs can be derived from various sources, including bone marrow, adipose tissue, embryonic tissue, cord blood, reprogrammed mature cells, and perinatal tissue. They support hematopoiesis and produce mesodermal cells. MSCs have demonstrated immunomodulatory and neurotrophic effects in animal models and early human trials.

As part of this study, Petrou et al. aimed to evaluate the safety and efficacy of repeated spinal injections of autologous MSCs in ALS patients. This open-label clinical trial included patients aged 20-70, with definite ALS diagnoses and ALS Functional Rating Scale Revised (ALSFRS-R) scores above 20. The patients received 1-4 intrathecal MSC injections at intervals of 3-6 months, with safety and tolerability as primary endpoints, and efficacy as secondary endpoints.

This trial found no serious adverse events, demonstrating the safety of repeated MSC injections. As evidence, the authors point out that, 15 out of 19 patients showed a reduction in the progression rate of their ALSFRS-R scores by more than 25% between the first and second injections, with an average improvement of 107.1%. Similar improvements were observed between subsequent injections. Thirteen patients experienced a 25% improvement in their progression rate over the entire treatment period, with an average improvement of 47.4%. Seven patients showed clinical improvement after the first transplantation, and five remained improved after the second cycle. These benefits were correlated with the intervals between the injections, suggesting that regular MSC administrations might be crucial for sustained efficacy.

Previous Studies on MSCs in ALS

Several small, open-label clinical trials have suggested that MSC treatment can be beneficial for neurological diseases, including ALS. In a phase I/II trial by the same research group, ALS patients received intrathecal and intravenous MSC injections, which were safe and showed a trend toward disease stabilization over six months. Another phase I/II and IIa trial with Brainstorm® used modified MSCs producing neurotrophic factors (MSC-NTF), showing at least a 25% improvement in disease progression, particularly in the intrathecally treated group.

Additional trials, including a randomized, placebo-controlled phase II study, demonstrated mixed results. While some trials noted improvements in a subgroup of rapid progressors, others did not show significant differences between MSC-treated and placebo groups overall. These studies highlight the need for repeated injections to maintain the benefits of MSC therapy.

Implications From the Current Study

According to Petrou et al., repeated intrathecal injections of MSCs over a longer follow-up period appears to induce significant, but short-term, clinical improvements and slow disease progression in a majority of patients. This study also reaffirmed the safety profile of MSC, with only mild and transient adverse events observed.

The study highlights the potential of MSC therapy in providing neuroprotection and slowing ALS progression. The immunomodulatory effects of MSCs, possibly reducing inflammation in the central nervous system, may also contribute to their therapeutic benefits. However, the small sample size and open-label design are limitations, necessitating larger, controlled trials to confirm these findings.

Future Directions

Petrou et al. concluded that repeated intrathecal injections of autologous MSCs are safe for ALS patients and suggest potential medium-term clinical benefits. However, larger studies are needed to confirm these findings. The consistent observation of safety and indications of efficacy across multiple cycles of treatment is encouraging, indicating that MSC therapy could slow the progression of ALS and improve patients’ quality of life.

The study’s promising results support the continued exploration of MSC therapy for ALS. The authors call for future trials to focus on optimizing the timing and frequency of MSC injections to maximize clinical benefits. Larger, controlled studies are essential to validate these findings and potentially establish MSC therapy as a viable treatment option for ALS. By addressing the unmet needs in neuroprotection and immunomodulation, MSC therapy holds the potential of improving the quality of life and survival for ALS patients.

Source: Panayiota Petrou, Ibrahim Kassis, Nour Eddine Yaghmour, Ariel Ginzberg, Dimitrios Karussis. A phase II clinical trial with repeated intrathecal injections of autologous mesenchymal stem cells in patients with amyotrophic lateral sclerosis. Front. Biosci. (Landmark Ed) 2021, 26(10), 693–706. https://doi.org/10.52586/4980

How Stem Cell Therapy Has Revolutionized Neurodegenerative Disease Management

How Stem Cell Therapy Has Revolutionized Neurodegenerative Disease Management

Neurodegenerative diseases, which include conditions like amyotrophic lateral sclerosis (ALS), motor neuron disease, Parkinson’s disease, and multiple sclerosis (MS), are characterized by the progressive loss of structure and function of neurons. These conditions are currently considered incurable and utilize treatments focusing primarily on managing symptoms rather than addressing the root causes. However, recent advancements in regenerative medicine, also known as stem cell therapy, particularly mesenchymal stem cell (MSC) therapy, have ushered in a new era of hope and potential for managing and potentially  these debilitating conditions. 

Understanding Mesenchymal Stem Cell Therapy

Mesenchymal stem cells (MSCs) are multipotent stromal cells capable of differentiating into a variety of cell types, including bone, cartilage, and fat cells. They can be derived from various tissues, such as bone marrow, adipose tissue, and umbilical cord blood. MSCs possess remarkable immunomodulatory and anti-inflammatory properties, which make them suitable for treating a wide range of medical conditions, including neurodegenerative diseases.

MSCs secrete a range of bioactive molecules that promote neuroprotection, neurogenesis, and angiogenesis. They can migrate to sites of injury or inflammation, where they modulate the immune response and promote tissue repair. Additionally, MSCs can differentiate into neuronal cells and support the survival of existing neurons by creating a favorable microenvironment.

Mesenchymal stem cells (MSCs) offer a multifaceted approach to managing neurodegenerative conditions with their unique properties and mechanisms of action. Here is how MSCs can help in neurodegenerative conditions:

1. Immunomodulation

MSCs have potent immunomodulatory effects, which can help in neurodegenerative conditions where inflammation and immune system dysregulation play significant roles. MSCs can:

  • Reduce Inflammation: By secreting anti-inflammatory cytokines, MSCs can reduce chronic inflammation in the central nervous system (CNS), which is a hallmark of many neurodegenerative diseases.
  • Modulate Immune Response: MSCs can alter the activity of various immune cells, including T-cells, B-cells, and macrophages, promoting a more balanced immune response and preventing autoimmune attacks on neural tissues.

2. Neuroprotection

MSCs can create a supportive environment for existing neurons, protecting them from further damage. They achieve this through:

  • Secretion of Neurotrophic Factors: MSCs secrete neurotrophic factors such as brain-derived neurotrophic factor (BDNF), glial cell line-derived neurotrophic factor (GDNF), and nerve growth factor (NGF), which support neuron survival, growth, and function.
  • Anti-apoptotic Effects: MSCs release molecules that inhibit apoptosis (programmed cell death), thereby preserving the existing neuronal population.

3. Neurogenesis and Differentiation

While MSCs themselves have limited capacity to differentiate into neurons, they can support neurogenesis indirectly:

  • Stimulation of Endogenous Stem Cells: MSCs can create a microenvironment that stimulates the body’s own neural stem cells to proliferate and differentiate into new neurons.
  • Paracrine Signaling: Through the release of various signaling molecules, MSCs can enhance the differentiation and maturation of progenitor cells into functional neurons and glial cells.

4. Tissue Repair and Regeneration

MSCs play a crucial role in repairing and regenerating damaged tissues:

  • Angiogenesis: MSCs promote the formation of new blood vessels, improving blood supply and oxygenation to damaged areas in the CNS, which is essential for tissue repair.
  • Extracellular Matrix Remodeling: MSCs secrete enzymes that remodel the extracellular matrix, facilitating tissue repair and regeneration.

5. Reduction of Oxidative Stress

Oxidative stress contributes to neuronal damage in many neurodegenerative diseases. MSCs can combat this through:

  • Antioxidant Enzyme Production: MSCs produce enzymes such as superoxide dismutase (SOD) and catalase, which help neutralize reactive oxygen species (ROS) and reduce oxidative stress.
  • Regulation of Oxidative Pathways: By modulating cellular pathways involved in oxidative stress, MSCs can protect neurons from oxidative damage.

6. Enhancement of Synaptic Connectivity

MSCs can improve neuronal communication and function by:

  • Promoting Synaptogenesis: MSCs secrete factors that encourage the formation of new synapses, enhancing neural connectivity and plasticity.

Supporting Synaptic Function: MSCs release molecules that help maintain and improve synaptic function, which is crucial for effective neural communication.

How Can Stem Cell Therapy Help Certain Neurodegenerative Conditions:

  • Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects motor neurons, leading to muscle weakness and atrophy. MSC therapy can help manage this condition by reducing inflammation and promoting the survival of motor neurons. Clinical trials have demonstrated that MSC transplantation can improve motor function and slow disease progression in ALS patients. The neuroprotective and regenerative properties of MSCs address both the symptoms and the underlying disease mechanisms, offering a potential option for those to consider.
  • Motor neuron diseases (MNDs) encompass a group of disorders characterized by the degeneration of motor neurons, leading to muscle weakness and paralysis. MSC therapy has emerged as a potential treatment for MNDs due to its ability to modulate the immune system and promote neuronal survival. Preclinical studies have shown that MSC transplantation can improve motor function and extend survival in animal models of MND. Ongoing clinical trials aim to evaluate the safety and efficacy of MSC therapy in patients with MND, offering hope for improved management and outcomes.
  • Parkinson’s disease (PD) is a neurodegenerative disorder characterized by the loss of dopaminergic neurons in the substantia nigra, leading to motor symptoms such as tremors, rigidity, and bradykinesia. MSC therapy has shown potential in PD treatment by promoting the survival of dopaminergic neurons and modulating the immune response. Preclinical studies have demonstrated that MSC transplantation can improve motor function and reduce neuroinflammation in animal models of PD. Clinical trials are underway to assess the safety and efficacy of MSC therapy in PD patients, with promising preliminary results. If successful, MSC therapy could offer a groundbreaking new approach to managing and potentially treating Parkinson’s disease.
  • Multiple sclerosis (MS) is an autoimmune neurodegenerative disease that affects the central nervous system, leading to a wide range of neurological symptoms. MSC therapy has shown promise in the treatment of MS due to its immunomodulatory and neuroprotective properties. MSCs can help reduce the autoimmune response, promote repair of damaged neural tissues, and improve overall neurological function. Clinical trials have indicated that MSC transplantation can reduce the frequency of relapses and slow the progression of MS, providing a new avenue of hope for patients who suffer from this chronic condition.

Advantages of MSC Therapy in Neurodegenerative Diseases

One of the significant advantages of MSC therapy is its low risk of causing immune rejection. MSCs are typically autologous (derived from the patient’s own tissues) or allogeneic (derived from a donor) and possess immunomodulatory properties. The anti-inflammatory effects of MSCs can mitigate the neuroinflammation commonly seen in neurodegenerative diseases, potentially slowing disease progression. 

MSCs can also promote neurogenesis and neuroprotection, supporting the survival and function of existing neurons and enhancing overall brain health. The ability of MSCs to migrate to sites of injury or inflammation allows for targeted treatment, maximizing therapeutic benefits while minimizing potential side effects.

Case Studies and Clinical Trials

Numerous clinical trials are currently underway to evaluate the safety and efficacy of MSC therapy in various neurodegenerative diseases, including ALS, MND, PD, and MS. Early-phase trials have shown promising results, with some patients experiencing improvements in motor function and quality of life. 

Case studies highlight the potential of MSC therapy to stabilize or improve disease symptoms, offering hope for patients with limited treatment options. The success of ongoing trials will provide valuable insights into the therapeutic potential of MSCs and pave the way for larger, more definitive studies.

The Potential of Mesenchymal Stem Cell Therapy in Neurodegenerative Disease Management

Mesenchymal stem cell therapy has revolutionized the management of neurodegenerative diseases by offering a novel approach to treatment that goes beyond symptom management. The ability of MSCs to modulate the immune response, promote neuroprotection, and support neuronal survival holds immense potential for conditions such as ALS, motor neuron disease, Parkinson’s disease, and multiple sclerosis. 

The remarkable properties of mesenchymal stem cells, including their ability to differentiate, migrate to injury sites, and modulate immune responses, make them a powerful tool in the fight against neurodegenerative diseases. As research progresses and our understanding deepens, MSC therapy could become a cornerstone in the treatment of neurodegenerative conditions, providing relief and improved quality of life for millions of patients worldwide. The journey towards fully realizing the potential of MSC therapy is ongoing, but the strides made thus far are a testament to the incredible possibilities that stem cell research holds for the future of medicine.

Stem Cell for Hair Loss

Stem Cell for Hair Loss

Stem Cell for Hair Loss Discover an alternative method to restore and regenerate new, healthier hair growth. Both men and women can experience hair loss, which can be caused by a variety of factors. The aging process affects every cell and organ in the body, and stem...
Regenerative Medicine for Hair Loss. Healthy Regrowth

Regenerative Medicine for Hair Loss. Healthy Regrowth

Your hair can be part of your identity. For some people, their hair is a symbol of their spiritual beliefs. Whether you’re focused on beauty and aesthetics or a deeper meaning behind your hair, hair loss can be devastating. 

For some people, regenerative medicine is an innovative, research-based solution to hair loss. Learn more about how regenerative hair loss treatments may restore your healthy locks. 

How Hair Loss Is Related to Your Well-Being  

Losing hair is often a symptom of an underlying health problem. While some people experience hair loss due to genetics or lifestyle choices, others may face lurking medical issues. Here are some of the ways your hair growth (or lack thereof) is related to your physical and mental wellness

Self-Esteem and Confidence 

You may feel embarrassed, ashamed, or self-conscious if you start to lose your hair. Both men and women struggle with remaining confident after significant hair loss. Your mental well-being may suffer if you can’t get your hair to grow back on its own. 

Some cultures associate losing hair with aging and other stereotypes. This can be devastating for someone going through medical, hormonal, or age-related hair loss. Healthy hair regrowth may help you regain the confidence you lost. 

Hormones 

Your hormones are chemical messengers that regulate all of your body’s processes. Hair growth is tied to your hormones, and any fluctuations or declines in certain hormones can lead to losing hair. Think about post-pregnancy hair shedding; this happens because your body’s cascade of pregnancy hormones changes. 

Hormonal changes as you get older can affect your hair. Age-related hair loss and thinning primarily affect women due to declines in estrogen production, but this problem can affect men as well. 

Genetic Baldness 

Male-pattern baldness is passed down through families. If you carry the gene for this condition, you may experience very early balding, starting in your 20s or 30s. This can change your appearance and make you feel older than you really are. 

There are no ill physical effects from male-pattern baldness besides losing hair. However, you may experience poor mental health as a result of your unwanted early hair loss and thinning. 

There is no cure for male-pattern baldness, but you can take steps to treat it. Regenerative medicine is here to intervene and treat hair loss in a natural, science-based way. 

ExogroPro: The Latest in Hair Regrowth Science 

When you experience hair loss of any kind, you probably have two priorities — prevent further hair loss and replenish your missing hair. ExogroPro, a professional-grade hair loss solution, addresses both of these problems at once. 

How It Works: Preventing Further Hair Loss and Regrowing Lost Hair

ExogroPro works to stimulate your hair follicles, causing them to grow healthy hair again. This treatment uses the power of specific exosomes, which carry materials between your cells. 

ExogroPro is designed to send signals to your hair follicles that stimulate hair growth. Because of exosomes’ extracellular communication abilities, they may be able to revive “dead” hair follicles on your head. 

Because of this treatment’s therapeutic effects on hair follicle health, it may help prevent further hair loss and thinning. By rejuvenating your hair follicles and preventing them from going dormant, ExogroPro offers a possible solution to unwanted lost hair. 

The Procedure: What to Expect 

The premium patent pending ExogroPro formula is injected into the scalp to deliver exosomes to the hair follicles. These exosomes originate from human stem cells, specifically mesenchymal stem cells. This is just another way we can harness the power of stem cells to regenerate and repair damaged tissues within your body. 

After two to six months, you may notice your hair growing back thicker and fuller than before. The results window varies, but one thing is certain — hair regrowth is worth the wait. 

The Benefits of Choosing Regenerative Medicine for Hair Growth

With many prescription hair growth drugs on the market, you may be wondering — why choose ExogroPro? Here are the major benefits of choosing regenerative medicine for hair regrowth and thickening. 

May Be More Effective Than Traditional Treatments

There are numerous treatments for hair loss, including PRP injections, topical oils, and more. These have varying efficacy and likely won’t give you the results you’re looking for. Prescription-strength hair growth drugs may not work for you either, resulting in frustration and discouragement. 

Regenerative medicine harnesses the power of science and innovation. By using human stem cells, we work with the body processes already in place within your system. Exosomes may prove to be more effective and efficient than other hair loss solutions. 

Drug-Free Therapy 

Exosomes are naturally found in the human body. When you choose ExogroPro, you choose drug-free natural therapy for your hair and scalp. 

While prescriptions can be helpful for some people facing hair loss, it’s understandable to want a natural alternative. We work with your natural healing and regrowth processes, not against them. 

Dual-Action Treatment: Prevention and Active Regrowth 

It’s impressive to consider how ExogroPro not only prevents further lost hair but can revive faulty hair follicles. This dual mechanism is key to getting the full head of hair you’re after. If it works for you, you’ll no longer have to mourn the hair you’ve lost — you could potentially get it back! 

Encourages Hair Health 

Solving hair loss isn’t just about replacing lost hair. It’s also about growing healthy, strong locks that can withstand damage and remain intact. ExogroPro promotes hair follicle health by improving cellular communications in the injected tissues. What’s more promising than the idea of a full, healthy head of hair after hair loss and thinning? 

Combating Hair Loss and Thinning with Medical Science 

It’s important to seek evidence-based treatments for your hair loss. No matter what the root cause of your hair problems is, you may find regenerative medicine to be a viable solution. 

You don’t have to mourn your lost hair forever. Regenerative medicine is at the forefront of medical innovation and science, which could spell good news for your hair. 

Everything You Need to Know About ALS

Everything You Need to Know About ALS

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease. Patients with this condition often experience a broad range of symptoms that get worse over time. While there’s no known cure, some interventions and regenerative therapies can be helpful.  

If you or a loved one has been diagnosed with ALS, it’s important to seek as much information as possible. That way, you can get the proper treatment and know what to expect as the disease manifests in your body. Here’s everything you need to know to be prepared for ALS. 

Lou Gehrig’s Disease: The Neurological and Muscular Effects 

ALS is also known as Lou Gehrig’s disease. Unfortunately, it has severe effects on your nervous system and muscular function as it progresses. 

Amyotrophic lateral sclerosis primarily affects your body’s motor neurons, which are responsible for dictating voluntary movements. Toward the end of the disease’s progression, involuntary movements like breathing can slow or stop entirely. 

When your motor neurons can no longer communicate with your muscles properly, your muscles start to waste away. This is called muscular atrophy. Once a muscle has atrophied, it no longer has enough tone to carry out normal movements. 

Since ALS is a progressive neurodegenerative disease, the symptoms get worse over time. In the later stages of the disease, ALS patients have trouble speaking, eating, swallowing, and making any voluntary movements at all. 

Who Gets ALS? Risk Factors and Genetic Components

Amyotrophic lateral sclerosis is usually a sporadic disease, meaning patients get it randomly without an obvious known cause. In some cases, although very rare, Amyotrophic lateral sclerosis is passed down through families. 

Genetic ALS stems from genetic mutations that are then passed on to children of affected parents. These cases only make up 10% or less of all ALS cases. 

Sporadic ALS may have certain risk factors, but there are no clear causes. This means scientists can’t yet point to a single cause of developing ALS if it’s not genetic. 

Possible Risk Factors for Sporadic Amyotrophic lateral sclerosis

Some loose associations between age, sex, and occupation have been made in relation to Amyotrophic lateral sclerosis cases. The presence of these risk factors does not guarantee that someone will develop ALS. 

These possible risk factors include: 

  • Age: Being 55 to 75 years old 
  • Sex: Early-life cases are most common in men 
  • Military service: Veterans may be at higher risk 

One possible reason that military veterans are at higher risk for Amyotrophic lateral sclerosis is because of their exposure to toxins like pesticides. Being around harmful chemicals may contribute to the development of this debilitating disease, but scientists aren’t sure of this. 

The First Signs of ALS

There are two types of ALS onset — limb onset and bulbar onset. Limb-onset Amyotrophic lateral sclerosis affects your arms or legs or both at the same time. You may notice problems with coordination, fine motor control, walking, and using your hands.

Bulbar-onset ALS first affects the neurons that control your speech and swallowing abilities. You might notice difficulty getting your words out properly or trouble swallowing. 

It doesn’t take long for the beginning symptoms of ALS to spread and get worse. This is often how physicians diagnose ALS, as rapidly progressing symptoms usually indicate a serious neurological problem. 

Progressive Problems and More Serious Symptoms 

As ALS develops, symptoms can become severe and even debilitating. Some patients experience changes seemingly overnight, while others develop more serious symptoms over weeks or months. 

The neuron degeneration caused by ALS can start to interfere with essential body functions such as breathing, blinking, and swallowing. 

Respiratory Symptoms

Respiration problems are common in end-stage ALS. The muscles in your chest that support breathing may become weak or paralyzed entirely. 

Some respiratory symptoms of ALS include: 

  • Shortness of breath (at rest) 
  • Excess saliva
  • Inability to clear your throat and lungs of mucus 
  • Pneumonia
  • Weak coughing 
  • Worsened breathing when lying flat 
  • Respiratory system failure 

Hospital interventions usually include ventilators to keep air flowing in and out of the patient’s lungs. 

Muscle Stiffness and Atrophy

As Amyotrophic lateral sclerosis interferes with their proper use, your muscles start to lose tone, mobility, and structure. This is known as muscle atrophy and may be accompanied by extreme stiffness. 

As Amyotrophic lateral sclerosis progresses, many patients lose the ability to speak loudly. The muscles involved in speech start to freeze up and become paralyzed. This can also cause an inability to swallow. 

Extreme Weight Loss

Being unable to consume food and fluids normally can lead to excessive weight loss in late-stage Amyotrophic lateral sclerosis patients. This isn’t a healthy type of weight loss, and it’s usually caused, in part, by loss of muscle mass. 

Mental Health Challenges 

Amyotrophic lateral sclerosis doesn’t affect your perception or cognition, meaning you can still hear, see, and think normally. Since the person is aware of their deteriorating condition, they may experience depression, anxiety, and other serious mental health challenges. Having a strong support system is key to remaining mentally well through your ALS journey. 

Treatment Options for ALS 

You can receive treatment for ALS to slow the progression of the disease. While there is no cure, medical intervention can help you maintain your quality of life for a longer period. 

ALS Medication

Some medications protect your motor neurons from further damage, which slows the progression of your ALS symptoms. These medications won’t revive damaged or dead neurons, but they can improve your disease prognosis. 

Physical and Occupational Therapy

Therapies that involve movement and motor functioning can help you maintain your muscle tone and avoid stiffness. While you still have the ability to move your limbs and smaller muscles, it’s crucial to practice doing so as much as possible. This repetition helps your brain and body stay in a good rhythm and slow motor damage. 

Regenerative Medicine

Some innovative treatments, like stem cell therapy for ALS, may improve your quality of life. Regenerative medicine focuses on healing and repairing damaged tissues and cells. 

Stem cell therapy uses unspecialized human cells to serve specific purposes throughout the body. In the case of ALS, stem cells are administered so they can differentiate into nerve cells to assist with your functioning and comfort. This may be worth considering if you want to approach your ALS symptoms from all possible angles. 

Living With Purpose: Life After an ALS Diagnosis

It’s important to maintain a positive outlook as much as possible. Having the support of medical professionals, family, and friends can make all the difference in your quality of life with Amyotrophic lateral sclerosis. Try not to lose hope; science is progressing toward new treatments every day. 

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