Amyotrophic Lateral Sclerosis (ALS)is a progressive neurodegenerative disease affecting nerve cells in the brain and in the spinal cord. As the disease progresses voluntary muscle action is severely compromised and the disease may cause total paralysis in its late stages.
Some genetic mutations can lead to inherited ALS, while patients with the non-inherited form of ALS display higher than normal levels of glutamate around the nerve cells in their spinal fluid. Glutamate is a chemical messenger in the brain. This chemical imbalance in the body, with glutamate at toxic levels, invariably leads to ALS.
A faulty immune system that attacks its own normal cells can cause the death of nerve cells also triggering ALS. Nerve cells may also be destroyed by the collection and accumulation of abnormal forms of proteins in the cells, again triggering ALS.
ALS Fact Sheet
Amyotrophic Lateral Sclerosis (ALS) is a neurological disease which impacts motor neurons that are involved in muscle movement throughout the body.
Heredity, genetics, exposure to toxic substances and smoking are some of the primary risk factors. Treatment for ALS has centred mainly on relieving symptoms and improving the quality of life for patients. This is referred to as palliative care and relies heavily on the services of occupational, physical and speech therapists. Social workers, nutritionists and diverse caregivers also play a key role in this palliative care. Home care is also critical for the well being of the patient. Under normal circumstances this palliative care only serves to alleviate the patient’s suffering. The progression of ALS invariably leads to death.
Promising ALS Research
Now, however, stem cells bring new hope for persons suffering from this deadly disease. In a study conducted by the University of South Florida, researchers were able to use bone marrow stem cell transplants to repair damage to the blood-spinal cord barrier in mouse models of ALS. The studies in the mouse models demonstrated improved motor functions and nervous systems conditions. If this is successfully replicated in humans, the implications will be wide ranging as palliative care takes a secondary role and real healing takes place.
The blood-spinal cord barrier is crucial in the development of ALS. This barrier is located between the blood circulatory system and the central nervous system. Researchers in the University of South Florida’s Centre of Excellence for Aging and Brain Repair see the repair of this blood-spinal cord barrier as a critical step in using step cells in the eventual treatment of human beings. This particular study is particularly significant because it used mice with ALS unlike previous tests which used pre-symptomatic mice. Using mice with the actual condition approximates as closely as possible the conditions (and possible reactions) of a human being with the illness.
To conduct this study, the scientists began by using stem cells harvested from human bone marrow and intravenously transplanted these into mice modeling ALS and which were already displaying clear symptoms of the disease. Four weeks into the treatment the transplanted stem cells succeeded in differentiating and attaching to the vascular walls of many capillaries. This effectively triggered the process of blood-spinal cord barrier repair. The scientists also observed that the results from the mice which received higher doses of stem cells were noticeably enhanced.
Stem Cells and ALS
Stem cells have the ability to differentiate. This is the capability of the cells to develop into practically any cell type in the body. This makes them ideally suited for use in restoring function that had been lost through neurodegenerative disorders (such as ALS) or through injury. These particular stem cells differentiated into endothelial cells. These are cells which make up the inner lining of a blood vessel thereby providing a barrier between blood and spinal cord tissue.
The result of this treatment was to delay the progression of the disease which resulted in improved motor function and increased motor neuron cell survival.
Spinal Muscular Atrophy (SMA) is a neurodegenerative disease targeting motor neurons. It normally occurs in the spinal cord. Nerve cells send messages from the brain to the muscles and vice versa. They are primarily responsible for bodily movements such as walking, swallowing, and breathing.
Severe strains of SMA cause paralysis and eventually death for the victims. Patients with milder forms normally experience weakness and wasting away of the muscular tissue. SMA involves the loss of nerve cells called motor neurons.
In most cases the proximal and lung muscles are affected first, but then spread to other body systems.
Neurons in SMA patients are unable to produce adequate amounts of a protein called Survival of Motor Neuron (SMN). This causes wasting away and eventually death of the cells.
Scientists have researched and developed different therapies to alleviate and treat the effects of SMA, and most of them have been targeted at fixing the gene itself. Approximately one in 50 people are carriers of the fatal genetic disease, which kills the most number of infants under the age 2.
Recent Harvard Research
In a recent discovery, researchers at the Harvard Stem Cell Institute (HSCI) have identified a compound that helps stabilise and protect the SMN protein. It uses induced pluripotent stem cells to make human models of neurological diseases.
Lee Rubin, a faculty member from the institutes’ Department of Stem Cell and Regenerative Biology sought to determine why motor neurons were targeted, and found out that the motor neurons experienced similar stress as those affected by Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease. His team of researchers also found out that some SMA affected neurons were dying before others, though they all experienced the same environment.
When the team analysed motor neurons derived from ALS patients, they found out that motor neurons with the highest levels of SMN protein were most likely to survive than those with lower levels. The research also showed that the survival of motor neurons depended on the availability of the SMN protein. The results suggest that if the amount of SMN protein is increased in any single motor neuron, the cell could be saved from dying.
In order to affirm these results, researchers led by Nadia Litterman induced human and murine motor neurons with a compound called Cullin, which is thought to regulate protein generation in cells. They found out that when exposed to Cullin, the SMA proteins became more stable and even multiplied abundantly. As a consequence of this, all motor neurons survived in the human specimens in the dish, and the in mouse models.
In summarising the results of the study, Rubin pointed out the discovery could end up benefitting patients who suffered from ALS as well as SMA.
Harvard’s office of technology development have filed an application to patent this approach towards the search for a cure for both diseases.
Amyotrophic Lateral Sclerosis (ALS) is a rare disease that mainly involve the nerve cells responsible for controlling the voluntary movement of muscles. ALS specifically attacks motorneurons in the brain stem, cortex and the spinal cord thereby causing effects such as muscle weakness, spasticity and paralysis. People affected by ALS experience difficulties in performing everyday functions like walking, breathing and talking. ALS is degenerative and it gradually causes a degeneration of motor neurons, leading to their death, and eventually leading to paralysis and death of the victim. This is usually within 3 to 5 years of initial diagnosis. Most of the victims of this disease die of respiratory complications as a result of the progressive weakening of the patient’s respiratory functions.
ALS Incidence
According to the Johns Hopkins Medicine, statistics show that ALS affects up to 30,000 patients in the United States of America with about 5,000 new cases being diagnosed every year. It is responsible for as many as five out of every 100,000 deaths of people aged 20 years or older. ALS indeed is a mystical disease as it is not easy to pinpoint the exact causes of ALS as it has no clear identifying cause. ALS does not impair a person’s intellectual reasoning, vision, hearing or sense of taste, smell and touch.
Amyotrophic Lateral Sclerosis (ALS) is a neurological disease which impacts motor neurons that are involved in muscle movement throughout the body.
ALS is also known as Lou Gehrig’s disease and has no known cure. It was first discovered by French neurologist Jean-Martin Charcot in the year 1869, but became more noticeable in the year 1939 after one of baseball’s most famous stars in the United States Lou Gehrig was diagnosed with it. Fans noticed that Lou Gehrig, once a very formidable baseball player had suddenly lost his touch and eventually pulled out of one of the games. He passed away barely two years after the first symptoms of ALS were noticed.
One of the most famous modern scientists, Prof. Stephen Hawking is known to suffer from the motor neuron disease. His doctors had believed that he would be dead within a few years. He requires a motorized wheelchair to move around as he is paralyzed. He also communicates through a special computer which he controls with a muscle on his cheek. Hawking’s case has defied a lot of theories on ALS as he is still alive almost 50 years after he was first diagnosed with it.
ALS can be further classified into two categories: sporadic and inherited. Sporadic cases of ALS occur in up to 90% of all reported cases, while about 10% is inherited from family members. The disease usually starts around the age of 60 and in inherited cases it starts at around the age of 50.
ALS Research
Research for the cure of ALS has been ongoing for quite a number of years. Scientists have proposed and tested many therapies and treatments, but the mortality rate due to effects of ALS still remains at almost 100%. All victims of ALS have succumbed to the debilitating disease.
The advancement of technology has enabled researchers to explore and study the disease, its effects and possible remedial cures to help decrease this mortality rate. Researchers have come up with Stem Cell-based Therapies that could eventually be used as a cure for ALS. This document will explore the use of Stem Cell-based therapies to treat the disease and also discuss what this advancement could mean for ALS patients in the near future.
Recently, Stem Cell-based Therapies, as potentially effective treatments of ALS, have emerged employing intraspinal, intrathecal, intramuscular, intracerebral, or intravenous autologous Stem cell administration routes.
Mesenchymal Stem cells
Mesenchymal Stem cells (MSCs) are multipotent adult stem cells that are present in all tissues. MSCs can differentiate into different tissues (meaning they can produce more than one type of specialised cell of the body) ranging from mesoderm, ranging from Osteocytes (bone cells), Chondrocytes (cartilage cells) and Adipocytes (fat cells). It has also been shown that MSCs could be pluripotent meaning that they can also differentiate into tissues and cells of non-mesodermal origin like neurons and epithelial cells.
MSCs can differentiate into many different types of cells that do not belong to the skeletal tissues, such as nerve cells, heart muscle cells and liver cells, which form the inner layer of blood vessels, and can therefore be used in the repair inured cell tissues of other cells of a different type.
Human undifferentiated Mesenchymal Stem Cells (hMSCs) can be harvested from different sources. They can be gotten from the bone marrow, the umbilical cord blood, adipose, and Wharton’s jelly. These cells have been tested in rodent models to treat diseases such as ALS and Spinal Cord Injury. Many studies have shown that the administration of human undifferentiated Stem cells is safe and can delay motor function and can increase the life expectancy of the subjected patients.
MSC and ALS Research
One test has increased worldwide interest in stem cell based ALS treatment. A group of scientists based in the Czech Republic performed a clinical trial in ALS patients to assess the safety and effectiveness of the administration of autologous bone marrow Stem Cell Therapy in a group of patients suffering from ALS.
In this particular test, bone marrow derived Mesenchymal Stem cells were used to assess the safety and efficacy of a single intrathecal administration of autologous Bone Marrow Mesenchymal Stem cells (BM-MSCs) in ALS treatment of patients with ALS. This trial was approved by the State Institute for Drug Control and the informed consent form approved by the ethics committee of the University Hospital Motol in Prague, Czech Republic.
All subjects entering the study gave an informed consent before any procedures specified in the protocol were performed on them. The patients were assured that the procedures involved in the study protocol would not interfere with the standard method of care and treatment.
A group of 26 people with symptoms of ALS were enrolled to participate in this program, and out of these 23 were found suitable for efficacy evaluation. These subjects were prescreened for a period of six months before Stem Cell Transplantation was performed. They were examined three times at 6, 3 and 1 month before treatment in order to check the progression rate of the disease. After that, they were also examined at regular intervals at 3, 6, 9, 12 and 18 months to assess the safety and effectiveness of the administered treatment.
Potential and adverse effects of this therapy were assessed during this period and the outcome was evaluated by the ALS Functional Rating Scale (ALSFRS).
The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) is an instrument for evaluating the functional status of patients with Amyotrophic Lateral Sclerosis. It can be used to monitor functional change in a patient over time. Such measures as speech, swallowing, handwriting walking and breathing are measured and compared over time.
The forced vital capacity and weakness scales of these patients were also assessed. Bone marrow derived stem cells were administered through a method known as intrathecal application where the bone marrow derived Stem cells are injected into the spinal theca. This is the area in the spinal cord that hold the cerebrospinal fluid. The implanted cells spread without crossing the blood-brain barrier.
Objectives of the Study
One of the primary objectives of this study was to assess the safety of the treatment to patients. All patients’ complaints regarding their medical conditions were recorded. They were monitored for reactions such as allergies, fever and any abnormal pains. Any bleeding, new infections and paralysis during the treatment were also recorded.
While in hospital, the patients’ temperature, blood pressure and any other vital functions were observed and recorded on a daily basis. If no further complications were observed the patients were discharged. 30% of these patients experienced mild to moderate headaches. No other serious adverse reactions were reported from this group.
The other objective of the trial was to evaluate the effect of the transplantation on the rate of disease progression pre and post treatment. The changes in ALSFRS after treatment were compared to the changes observed prior. The result of this was that there was a significant stabilisation of ALSFRS score in the patients. Tests after 6 months showed a marked slowdown in disease progression.
Conclusion from the Study
From the results of these tests, it can be concluded that intrathecal application of BM-MSC is ALS patients is a relatively safe procedure and can slow down the progression of the disease.
It also might be important to start the therapeutic intervention much earlier, similar to animal models, but this would require earlier ALS diagnosis and/or identification of early disease markers in suspected cases. There is a need for further clinical trials to elucidate the most effective cell type, the most effective methods of delivery, and proper doses in single or repeated applications.
Such positive results show that a lot of progress has been made show that ALS can be contained and this gives a lot of hope to the victims of ALS in the coming future.
Stem Cell Treatment for ALS Available Today
We are excited to offer Stem Cell Therapy at our state of the art facilities to stop the progression of ALS in our patients.
We have a dedicated team of highly skilled doctors, nurses and other practitioners based in our world class medical facility in Bangkok, Thailand. They work around the clock to ensure our patients comfort and welfare before, during and after treatment is of world class standard.
Our patients, who come from around the world, are a testimony to the fact that they have been able to notice a change in the way their bodies respond to treatment. To ensure that our patients get the best out of the Stem Cell-based Therapy, we administer a combination of supportive remedies such as injection of growth factors and vitamins, rehabilitation and detoxification programs to treat them.
Amyotrophic Lateral Sclerosis (ALS) (also known as Lou Gehrig’s disease, and named after the celebrated baseball player) is a neurological disease that targets the nerve cells (or neurons) which are responsible for controlling voluntary muscles in the limbs and face, among other organs. It is a rapidly progressive disease and was first identified by the French neurologist Jean-Martin Charcot in 1869.
It however only came to both national and international prominence when it prematurely ended the baseball career of Lou Gehrig, the New York Yankee’s Hall of Fame first baseman, in 1939. Not surprisingly the United States government provides the largest proportion of funding in the research of this disease. Although federal support has been consistent sustained advocacy ensures that more funding is forthcoming in the effort to find a cure for ALS.
ALS Research
Scientists whose work involves research on ALS have access to the National ALS Registry which is congressionally mandated to catalogue information on persons in the U.S. living with ALS. About 30, 000 patients in the country are living with the disease with 5,000 new diagnoses every year.
ALS Early Warning Signs
The early warning signs of ALS include general clumsiness which is characterised by tripping and falling, difficulty in walking and performing normal chores and hand weakness. Muscle cramps in the arms, shoulders and tongue are also among the early symptoms associated with ALS. Difficulty in swallowing and slurred speech may also indicate the onset of ALS. The inability to maintain good posture and to hold one’s head up are typical early warning signs of ALS. Weakness in the legs, feet and ankles may also be an indication of the presence of ALS.
It is important to note that ALS will invariably start in the limbs and progressively spread to other parts of the body. This advance is what eventually affects speaking, breathing, swallowing and chewing. One positive characteristic of ALS is that it very rarely infringes on bladder and bowel control and thinking ability.
Up to 10 per cent of all known cases of ALS are inherited. The remaining 90% have no known origin although scientists are following up on several leads that they believe will eventually yield results.
Amyotrophic Lateral Sclerosis (ALS) is a neurological disease which impacts motor neurons that are involved in muscle movement throughout the body.
ALS Risk Factors
Risk factors generally associated with ALS include age, with the common age bracket under threat being the 40 to 60 years. Heredity is another established risk factor with up to 10 % of reported cases displaying this feature. Other risk factors include smoking and exposure to lead and other harmful substances. Persons who have seen military service appear to be more predisposed to acquiring ALS. Studies have yet to pinpoint what it is in the military that triggers the onset of ALS. This may be one or more of the various activities associated with the armed forces which include intense exertion, traumatic injuries and exposure to certain metals or chemicals among other factors.
An absolute cure for ALS is yet to be found. What are currently available are treatments to control the symptoms while preventing unnecessary complications and easing the lives of those living with the disease.
ALS Awareness Month will be a time for reflection, taking stock of gains achieved and exploring what the future holds in terms of treatment for this deadly disease.
Amyotrophic Lateral Sclerosis (ALS) has been a particularly troublesome disease to manage due to the failure of growth factors in clinics. ALS is a neurological disorder that affects nerve cells (or neurons) that are responsible for controlling voluntary muscle movement. Specific types of these motor neurons in the spinal cord rely in part for their survival on distinct growth factors. Georg Haase of Aix-Marseille University in France led a study whose results suggest that when these growth factors are combined they may afford protection to motor neurons that have been damaged by disease.
The potential benefits of growth factors in managing ALS been known to the medical profession for decades now. In the early 1990s neurotrophic factors (NTFs) which are peptides (or small proteins) and which are a family of biomolecules that support the growth, survival and – crucially – the differentiation of developing and mature neurons were used in early therapies to control ALS. These therapies were however ineffective and it is only in recent years that researchers have found that when these growth factors are combined then the expected results are more assured. The combination of these growth factors provide trophic (nutrition-related) support for motor neurons, especially in the developing spinal cord.
What is ALS?
Amyotrophic Lateral Sclerosis (ALS) is a neurological disease which impacts motor neurons that are involved in muscle movement throughout the body.
A working hypothesis that seeks to explain why a combination of growth factors seem to work (as opposed to when only one type is used) postulates that particular types of motor neurons are protected by particular types of growth factors. Indeed Georg Haase and his team, through rigorous scientific processes have put this theory to the test to ultimately identify which particular NTFs protect which distinct classes of motor neurons in the developing lumbar spinal cord. Using this technique researchers will be able to isolate the substances needed to protect adult motor neurons and those that have been damaged by ALS. The results of these extensive analyses will have wide-ranging implications in the therapies used to control ALS as neuroprotective treatment strategies will be tailored to the disease.
Further the use of Mesenchymal Stem Cells (MSCs), when combined with a spinal tap or lumbar puncture (LP), enhances the levels of many of these growth factors. MSCs are multipotent stromal cells which have the capability to differentiate into many different cell types and are therefore are a rich source in the treatment of a variety of disorders. MSCs can differentiate into adipocytes, which are fat cells, bone cells (known as osteoblasts), myocytes – which are muscle cells – and cartilage cells, known medically as chondrocytes.
Stem Cell Therapy to manage Amyotrophic Lateral Sclerosis is already in use and the benefits that accrue from this treatment are worthy of note. Stem Cell Therapy has been shown to alleviate the severe symptoms that characterise ALS. Patients have reported improvement in many areas including improved speech, easing of neuropathic pain and fatigue and amelioration of motor function. Patients have further witnessed an improvement in balance and coordination, the diminishing of tremors, easier swallowing and a general slowing down of the disease.
Former 49ers wide receiver legend Dwight Clark has announced via Twitter that he is suffering from ALS, also known as Lou Gehrig’s disease.
What is ALS?
Amyotrophic Lateral Sclerosis (ALS) causes motor neuron degeneration. As we know, nerve cells control voluntary muscle movement, and when this disease attacks, victims will gradually experience symptoms such as difficulty in speech, swallowing, tripping and even breathing difficulties
Amyotrophic Lateral Sclerosis (ALS) is a neurological disease which impacts motor neurons that are involved in muscle movement throughout the body.
Approximately 6,000 people in the USA alone are diagnosed with ALS every year.
Dwight Clark announces he is suffering from ALS
Football players and fans are offering support to 49ers legend Dwight Clark. One of the fans who is suffering from ALS has offered his encouragement.
Matt Chaney from Lafayette was diagnosed with ALS in 2001. His ALS is progressing very slowly. He uses a wheelchair to get around and an Ipad to communicate when his speech is fatigued.
One of the many challenges of ALS is the loss of independence, of which Tami, Chaney’s caretaker helps him with.
As the 49ers great Dwight Clark faces this disease, Dr. Jonathan Katz of California Pacific Medical Center offers some hope.
ALS Research
“Scientific research is really reaching a point right now where things are beginning to tip right now. We’re getting a better understanding of the disease,” said Dr. Katz. Dwight Clark, who played for the San Francisco 49ers team of the National Football League from 1979 to 1987 says he believes there’s a link with football. Mr. Chaney isn’t sure though, he also played college football back in the 1970s.
With proper funding of research to find a treatment and a cure, Chaney is hoping for some conclusive answers.
As much as Chaney, a married father of two is able to still do, he is the first to admit that ALS is a nightmare and he devotesmost of his free time to raising awareness and funds for a cure. He has been recognised for his work with the ALS Association, and is one the board of directors for the Golden West Chapter of the ALS Association.
He has offered to share his experiences with Dwight Clark and others who has been affected by this debilitating disease.
“If they have any questions he’s here,” said Tami, Chaney’s caretaker. Some excerpts from Clark’s letter:
“In September of 2015, I started feeling weakness in my left hand. I was mildly paying attention to it because since my playing days, I’ve constantly had pain in my neck. I was thinking it was related to some kind of nerve damage because it would just come and go.” “…What I do know is I have a huge battle in front of me and I’m grateful for the strength and unconditional love from my wife Kelly..She keeps thinking positive and convinces me each day that we can beat this, as does my daughter Casey and my son Mac…been unwavering with their love and support. My ultimate hope is that eventually I can assist in finding a cure for ALS, which disrupts the lives of so many and their loved ones.”
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