No Known Cause or Cure
Today, there are approximately 30,000 Americans living with ALS, and every year, 5,000 more will be diagnosed. Studies have shown that 90 percent of ALS cases are sporadic, which means the individual did not inherit the disease. Because the potential causes of the disease are so diverse, it is challenging to find a therapy that would work for everyone. In fact, only in the last few years have they actually been able to test if a drug would work on motor neurons.
There are currently two FDA-approved ALS drugs in the United States and they are riluzole and edaravone. Unfortunately, neither one of these drugs is considered an effective treatment. Evidence shows that they do slow disease progression in some patients, but it’s only by a few months.
Stem Cell Therapy For ALS
By using stem cell therapy for ALS, there is hope that a treatment plan or cure can be created. Given the success of stem cell therapy in treating other conditions, there is hope that ALS can be added to the list. Organizations like the Harvard Stem Cell Institute have been researching the possibilities and have discovered helpful information.
Advances in stem cell biology has permitted the production of millions of motor neurons created from mouse embryonic stem cells obtained from ALS genetic disease models. Scientists have also been able to obtain human induced pluripotent stem cells from the blood and skin of ALS patients. This discovery means that ALS can be studied in a laboratory culture dish filled with the cells that cause this condition. This allows scientists to explore new therapies for ALS, and potentially a cure.
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Induced Pluripotent Stem Cells and ALS
Scientists have found that glial cells actually weaken the motor neurons in patients with ALS. This weakening results in uncontrolled movements and muscle weakness in patients with the disease. By using induced pluripotent stem cells to recreate glial cells and motor neurons, scientists can screen if drugs actually change the disease process. The process is actually quite unique. The motor neurons created from induced pluripotent stem cells are genetically modified to create fluorescent markers that allow a clear view under a microscope. This allows scientists to track the motor neurons over time and gather an understanding of if certain treatments work. Getting this type of research was simply not possible prior to stem cell research.
Providing An Unlimited Supply of Cells
The use of induced pluripotent stem cells gives scientists the ability to make an unlimited supply of cells to study in a dish; this is groundbreaking. By taking a skin or blood sample from a patient with ALS, scientists can create cells that match an individual’s genetic makeup. Scientists can really study the disease process and ask questions like:
- What is unique about this case?
- When was disease onset?
- What is common about this case?
- Has the severity of the disease changed? If so, what could have triggered it?
How Would Stem Cell Therapy For ALS Look?
According to the ALS Association, the most common application of stem cell therapy will likely be either using stem cells or obtaining cells from the patient and having them deliver protective molecules to motor neurons in the spinal cord. They also envision doing a transplantation of astrocytes, which are support cells, to assist the motor neurons in the brain and spinal cord.
The ultimate goal is to replace failing motor neurons with new and healthy ones. However, we still have quite a ways to go. Instead, researchers will focus on using stem cells to deliver neurotrophic factors. By doing this, they’ll be able to observe if and how neurotrophic factors can effectively support the growth of neurons.
Clinical Trials And Stem Cell Therapy For ALS
To date, there is a clinical trial that reviews the effects of retigabine on motor neurons. Induced pluripotent stem cells are obtained from skin samples of patients with ALS and they test if there is a change in the excitability of motor neurons in people with ALS.
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What About Mesenchymal Stem Cells?
Although induced pluripotent stem cells are the most researched for ALS, there are also mesenchymal stem cells. This form of therapy involves taking marrow or fat tissue from a patient’s bone and then altering it to boost production of neurotrophic factors. The goal is to have the neurotrophic factors protect the very neurons that are destroyed by the disease. In addition, researchers would like the stem cells to develop into healthy motor neurons.
Some Treatment Centers Boast A Cure
Many clinics out of the country, and even in the United States, prey on vulnerable individuals looking for a last hope. They have sleek website designs and convincing online advertisements, but they don’t actually have the cure. If you or someone you know is looking for a treatment center, then find one that is FDA-approved. These FDA-approved centers usually have clinical trials that patients who meet certain criteria can participate in.
There’s Hope In Stem Cell Therapy For ALS
Stem cell therapy is popping up all over the place as the “cure all” of the future, and for a very good reason. There is hope that the clinical trials being done today can find the cure for tomorrow. Fortunately, there is promise in stem cell therapy for ALS. Finding a cure would change the lives of the more than 30,000 people who currently struggle with the progressive disease, and their families.