Although records of ALS aren’t kept everywhere in the country, doctors estimate that between 12,000 and 15,000 people in the United States have it. Though this nerve disease is relatively rare, the illness has a devastating, paralyzing effect on the people who have it.
As of now, ALS isn’t curable. However, many clinical trials show promising results in treating it in the future. Among these trials, the use of stem cells for ALS stands out as an innovative solution.
In this article, we’ll cover everything you need to know about where stem cell therapy stands as a possible solution to Lou Gehrig’s disease. Let’s get started!
What Is ALS?
The disease known as ALS stands for amyotrophic lateral sclerosis. It’s a condition that affects the motor neurons in our bodies that are responsible for voluntary muscle movements. The progressive loss of these motor neurons leads to muscle twitching, muscle wasting, and difficulty speaking.
Eventually, the disease causes paralysis and early death caused by respiratory failure. The worst part is that around 95% of the cases have no known cause. This makes ALS incredibly difficult to study.
How Can Stem Cell Therapy Help ALS?
There are a variety of stem cells that come from different sources. For the sake of ALS, you’ll need to understand embryonic stem cells (ESCs), induced pluripotent stem cells (iPSCs), and adult stem cells, such as mesenchymal stem cells, for example. We’ll briefly go over these types, but if you want to learn about the others, then make sure to check out our guide.
Embryonic stem cells (ESCs) are stem cells that are derived from early stage human embryos. This makes them controversial. It also means they can become any of the 200 different cells types that form a grown human. Theoretically, these cells have the capacity to replaced damaged or diseased tissue, which makes them a promising cell type for the treatment of ALS.
Induced pluripotent stem cells are typically made from adult skin cells (fibroblasts) or blood cells. These cells are treated in a lab to cause them to revert to an embryonic stem cell like state. They are not controversial, because they are derived from differentiated adult cells.
Adult stem cells are stem cells present within various tissues of a live human being, regardless of age. This makes them non-controversial.
Mesenchymal stem cells are a type of adult stem cell that is commonly used for therapeutic purposes. They are found in high quantities within the bone marrow, fat (adipose) tissue, dental pulp, and other bodily regions.
So, how do stem cells help with ALS?
One of their key uses right now is to help researchers understand more about the disease. Scientists have been using iPSCs from patients with ALS and differentiating them into diseased motor neurons. This approach is sometimes called making a “disease in a dish” (a lab dish, that is).
These motor neurons reveal a lot about why the original motor neurons die due to the disease. The great thing about iPSCs is that they’re a snapshot of a person’s genetic material. They can also be replicated for an unlimited amount of time to be studied.
In a rare disease like ALS, this is invaluable. However, stem cells aren’t just important for studying. They also may hold the key to a potential cure. Potential approaches involve injecting either stem cells, the cells they have been differentiated into (such as motor neurons), or the factors they secrete into the brain or spinal cord of a diseased patient.
In theory, these cells may help to protect the decaying motor neurons found in ALS patients. However, it’s important to remember that these types of treatments are still in early clinical trial stages.
Understanding Clinical Trials
It’s important to distinguish between real types of stem cell research and unapproved clinics. There are many deceptive practitioners who mislead desperate ALS patients into thinking they can get a cure in exchange for money.
However, depending on the stage of a person’s disease, even unvalidated treatments might make sense in some cases. This statement assumes that the treatment is administered by a reputable stem cell treatment provider who closely adheres to the regulatory protocols within their region.
In reality, stem cell clinical trials require a lot of testing with few guarantees of success. For a treatment to be legitimate, it must go through four distinct clinical phases.
The first of these phases is the pilot trial, which is performed to ensure that the drug or treatment is safe and viable. After that comes phase one of a clinical trial, this type of trial is typically limited to small sizes of twenty people. During this stage, researchers are looking for side effects.
If anything dangerous occurs, then research is immediately ended. Next comes phase two. Patient sizes for phase two are typically larger — around one hundred people — to determine the optimal dosage level.
Finally comes phase three. During this final phase, the sample size is expanded to include hundreds of patients. The treatment is then determined to be either statistically effective or ineffective.
Because of the rare nature of ALS, clinical trials are slow to progress. However, there are a few that are currently in the works.
Clinical Trials Using Stem Cells for ALS
Ten years ago, an investigative treatment known as NurOwn began injecting mesenchymal stem cells into the spinal cord and muscles. The goal of which was to harness the neurotrophic factors secreted by the cells. The initial review of the phase 1/2 and 2a clinical trial proved that the method was safe.
On top of that, it was shown to slow the progression of ALS in patients six months after the injection. NurOwn is now moving on to a phase 3 trial that’s in the works to assess the efficiency of the treatment.
Another new study is using astrocytes that come from human embryonic cells to treat the disease. The cells, which they refer to as AstroRx, show promise in riding the body of excessive glutamate and reduce the amount of oxidative stress and other toxins that accompany ALS.
Stem cells are also being used to test other types of medicines. For example, treatments like Retigabine use iPSCs from patients to determine whether the test is having the desired effect.
The Future of Stem Cells for ALS
Hopefully this article helped you learn about the current wave of research surrounding stem cells for ALS. Ultimately, ALS is one of the most difficult diseases to treat.
Not only is it rare, but it differs tremendously from patient to patient. However, the power of stem cell therapy holds intriguing potential. It’s too early to tell, but promising clinical trials are already underway.
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