For those who are suffering from Lou Gehrig’s Disease — now commonly referred to as Amyotrophic Lateral Sclerosis (ALS) — finding treatment for the condition can seem like a never-ending quest. Stem cells have been used for several years to treat a wide variety of diseases, and many of these treatments point to the potential for further exploration.
Some of the beneficiaries of this process are ALS sufferers. Although there is no cure for ALS, stem cell therapy offers the chance to slow the progression of the disease while also helping to control its symptoms. While there are currently drug treatments available to address the disease, they all have side effects and may offer only limited means of controlling ALS.
In contrast, regenerative medicine, also known as stem cell therapy, offers a new avenue for pursuing ALS treatment, one that shows promising potential for improving patient outcomes over the long term. Regenerative medicine provides patients another path to managing their condition, one that may provide improved symptoms and potential long-term benefits.
What are Stem Cells?
The primary cells used for ALS-related stem cell therapy are called mesenchymal stem cells. These cells are derived from adipose (fat) or umbilical cord (Wharton’s Jelly) cells, and they have a unique ability to differentiate themselves into a wide variety of tissues.
Once inserted into the human body, the goal is to have them protect against cell loss by regrowing nerve cells and pathways in the brain.
Stem cell procedures like the ones used for treating ALS are generally considered to be safe, as patients rarely suffer from complications or side effects. In a study published in Neurology, for example, patients treated with mesenchymal cells did not show a markedly elevated risk for complications.
While stem cells for ALS patients should not be seen as a potential cure, many patients have reported improvement in some areas, including:
Repairs in nerve damage
Reduced progression of the disease
Motor skill improvements
Higher energy levels
While these results can’t be guaranteed for all patients, they represent major steps forward for those who have not had success with traditional treatments or those who need more help in dealing with progressively worse symptoms of ALS.
Like many treatments for ALS, stem cell therapy is considered to be experimental, which means that it does not have approval from the FDA. However, regenerative medicine may offer a window to an improved patient experience by easing symptoms of the disease that would otherwise be debilitating. If you would like to learn more or schedule a consultation, contact a care coordinator today!
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare disease in which the body’s neurons that control voluntary muscles begin to degenerate. Patients experience muscle weakening and involuntary spasticity, as well as symptoms such as muscle cramps and stiffness, and eventually, difficulty moving, speaking, swallowing, and breathing.
While there is currently no cure for the devastating illness, there are drugs that can increase the quality of life and marginally slow the disease’s progression. Researchers have long been pursuing a more effective treatment for the disease, and efforts were increased significantly as a result of the 2014 viral Ice Bucket Challenge, which raised at least $115 million for research efforts.
Stem Cell Therapy for ALS
One area that’s of particular interest to researchers is regenerative medicine therapy. Also known as stem cell therapy, this option could be a potential treatment for ALS, as it could help sustain and nurture motor neurons that have been compromised by the disease. This is due to stem cells’ ability to release neurotrophic factors, which support and protect nerve cells. This is not a cure, nor a guarantee, but is an option to help slow down the progression of the condition.
Stem cells can be harvested from sources such as the umbilical cord (Wharton’s Jelly), or the patient’s own adipose (fat) tissue, then strategically transplanted at locations such as the spinal canal, intravenous, or muscle tissue. Once in the brain tissues, stem cells have the potential to protect healthy neurons and replace those that have been compromised.
Experts are using stem cells both for research purposes, by creating cells genetically identical to patients to see how they’ll respond to treatments, as well as for treating patients directly. With their protective qualities, the cells can help preserve healthy cells and repair or replace those that have been damaged.
According to results from clinical trials, 87% of patients who received the treatment responded to the treatment with at least 25% improvement and slowed disease progression. Evidence also suggests the treatment is safe and well-tolerated.
While much of how ALS develops remains a mystery, researchers are hopeful that further investigation into stem cell therapy will help to drastically improve treatment outcomes compared to the drugs currently available. If you would like to learn more contact a care coordinator today!
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease or Lou Gehrig’s disease, is a disease that gradually paralyzes people because the brain is no longer able to communicate with the muscles of the body that we are typically able to move at will; as ALS progresses, people will lose the ability to walk, talk, swallow, and eventually breathe.
While no treatment to prevent or even slow the progression of, ALS currently exists, recent findings indicate that neurotrophic factors (NTFs) have been shown to potentially improve the survival of motor neurons in ALS. While a single administration of NTFs has not been effective in extending the life of these motor neurons, the review suggests the direct delivery of multiple NTFs by transplantation to the CNS has proven effective in animal studies.
Specifically, the observed benefits of mesenchymal stem cells (MSC) transplanted from bone marrow or adipose suggest improved neurological stabilization in patients with ALS. As such, the authors of this review have developed a method that produces a strong synergistic effect when introducing a combined delivery of neurotrophic factors in patients with ALS.
The authors, in this review, report on the safety and clinical effects resulting from phase 1 / 2 and 2a clinical trials in which autologous MSC-NTS cells were transplanted in patients with ALS. Both of these studies were considered open-label proof of concept studies where patients were followed up for 3 months before transplantation and 6 months after receiving MSC-NTS transplantation.
No serious adverse events were associated with MSC-NTF cells intramuscular (IM) injections, intrathecal injections (IT), or a combination of both (IT+IM) during these studies.
Additionally, neurotrophic growth factor secretion of patients’ cells was shown to be induced in the MSC-NTF cells when compared to MSCs of the same patient prior to differentiation. In all samples, MSC-NTF cells demonstrated increased secretion of NTFs when compared to non-differentiated MSCs from the same patient.
As a result of this study, the authors have concluded that IT and IM injections of MSC-NTF cells in patients with ALS are safe and well-tolerated. While not the primary focus of the study, the findings also demonstrated clinically meaningful benefits specifically induced by intrathecal treatment with MSC-NTF cells, including potentially slowing the rate of ALS progression.
Considering that neurologists specializing in the treatment of ALS consider a reduction in ALS-FRS-R slope of 25% or more to be clinically significant, the change in ALS progression rate observed after MSC-NTF cell transplantation in this study may indicate a clinically meaningful effect to be confirmed in future clinical trials.
ALS, which stands for amyotrophic lateral sclerosis, is a neurodegenerative disease that has no cure and no substantially effective treatment. Today, there are two drugs available that can slow the progression of ALS— riluzole and edaravone—but these agents may only be modestly effective. Sadly, patients with ALS get progressively worse, sometimes even with treatment. They lose to ability to move, to swallow, and to breathe. It is a devastating disease.
As with other neurodegenerative diseases, researchers are trying to treat ALS with stem cells. Stem cells have the capacity to become other types of cells such as neurons or glia. The notion is that stem cells could be used to rescue and replenish the nerve cells that are dysfunctional or destroyed in ALS. Studies are emerging that suggest that these approaches may bear fruit.
One such stem cell trial comes out of India, published by Dr. Prabhakar and colleagues. The research scientists infused autologous bone marrow-derived stem cells (autologous means the cells were retrieved from the patient and then readministered to the same patient). The scientists conducted the clinical study in ten patients with ALS who had a mean revised ALS Functional Rating Scale score of 30.2 ± 10.58. In other words, patients with this score have about a 60 to 70% chance of being alive for 9 more months.
Impressively, patients treated with autologous bone marrow-derived stem cells did not have a significant reduction in ALS Functional Rating Scale score after one year, which means their disease stayed relatively stable over this time instead of deteriorating. It took about 16.7 months for the score on this scale to drop by 4 points, which is considered a significant drop. The median survival after the procedure was 18.0 months, which is substantially more than would have been expected at the start of the study.
Taken together, these results suggest that patients with moderately severe ALS enjoyed a stabilization of their ALS symptoms for over 16 months on average. While there was no placebo control group, the stem cell-treated patients survived about twice as long as was estimated at the start of the trial.
These positive results must be confirmed in a larger, placebo-controlled trial. However, they strongly argue for further research of stem cells for the treatment of amyotrophic lateral sclerosis.
Reference: Prabhakar S, Marwaha N, Lal V, Sharma RR, Rajan R, Khandelwal N. Autologous bone marrow-derived stem cells in amyotrophic lateral sclerosis: A pilot study. Neurol India 2012;60:465-9
Amyotrophic lateral sclerosis is a cruel disease. It causes the nerves that control muscles to die. When these upper and lower motoneurons degenerate, it causes weakness, muscle atrophy, muscle cramps, and twitching. Patients with progressed ALS lose the ability to walk and to move, and ultimately lose the ability to swallow and to breathe.
Unfortunately, there is no cure for this disease. Two drugs, riluzole, and edavarone, can help slow the progression of amyotrophic lateral sclerosis; however, these treatments cannot stop the disease.
Stem cells are an intriguing potential option for those wanting to manage symptoms from neurodegenerative diseases like ALS. The hypothesis is simple. Scientists are aggressively pursuing stem cell research to treat amyotrophic lateral sclerosis to study the use of stem cells to potentially restore diseased nerve cells in ALS which may help to restore muscle function. Drs. Gugliandolo, Bramanti, and Mazzon recently reviewed the potential use of mesenchymal stem cells for the treatment of ALS.
Mesenchymal stem cells can be gathered from many different sites in the body including bone marrow, umbilical cord, or adipose (fat) cells. The stem cells can then become several different cells in the body, including nerve cells (i.e. neurons). Mesenchymal stem cells also produce and release (e.g. through exosomes) an astounding number of molecules that help other cells grow and develop. Thus, mesenchymal stem cells can not only become new nerve cells, they can support other nerve cells’ growth and development.
The authors describe in detail the potential for mesenchymal stem cells to help treat ALS. The review shows the many successful uses of stem cells in animals (mice) that have experimental ALS. In short, stem cells slowed the loss of motor function (muscle activity), delayed the progression of ALS, and increased length of survival.
Clinical trials of mesenchymal stem cells to treat ALS are in Phase I and Phase II, however initial results are encouraging. First of all, treatment with these stem cells is safe in patients with ALS—no serious adverse events have been reported in any of the trials reviewed in the journal article. In at least 9 clinical trials, mesenchymal stem cells slowed disease progression in patients with ALS to some degree.
Patients with ALS and those who care for them should note that while these trials have shown that mesenchymal stem cells are safe and at least partially effective in the treatment of ALS, Phase I and Phase II clinical trials only have a relatively small number of patients compared to Phase III trials. Nonetheless, the clinical trial results thus far look promising, certainly promising enough for certain types of stem cells to advance to Phase III pivotal clinical trials.
Reference: Gugliandolo, A., et al. Mesenchymal Stem Cells: A Potential Therapeutic Approach for Amyotrophic Lateral Sclerosis? Stem Cells International. Vol. 2019, Article ID 3675627, 16 pages, 2019. https://doi.org/10.1155/2019/3675627
ALS, also known as amyotrophic lateral sclerosis or Lou Gehrig’s disease, is a progressive neurologic condition. There is no current cure for this condition and the only treatments, riluzole and edaravone, are only modestly effective at slowing the disease.
This lack of a cure or effective treatment for ALS is not for a lack of trying. There have been countless clinical trials to test treatments for ALS—almost all have ended in failure in Phase 1 or Phase 2 clinical trials. Indeed, few potential ALS treatments make it to Phase 3 trials—the final stage of clinical testing before a drug is approved by the FDA for treatment.
It is this history of failure that has sparked so much enthusiasm in a new stem cell treatment for ALS. The potential stem cell treatment for ALS is currently known by its development name, NSI-566. NSI-566 is made up of neural stem cells—cells that can become new motor neurons. NSI-566 is injected into the spinal column of patients with ALS so that the stem cells can hopefully restore motor function and improve ALS symptoms.
The Phase 1 study showed a spinal injection of NSI-566 was safe in ALS patients, a finding that was confirmed in the Phase 2 trial. Perhaps more importantly, results from the Phase 2 trial showed that patients who received stem cell treatment did substantially better 24 months later than those who received a placebo. These results were so encouraging, that NSI-566 now heads to Phase 3 clinical testing.
While it will take some time to conduct the Phase 3 clinical trial, reaching this milestone is an achievement in itself. Any Phase 3 trial design will require a relatively large number of patients, so those who are interested should occasionally check clinicaltrials.gov or speak to your neurologist about enrolling as a volunteer.
Reference: Goutman, S., et al. (2018). Long-term Phase 1/2 intraspinal stem cell transplantation outcomes in ALS. Annals of Clinical and TranslationalNeurology 2018; 5(6): 730–740
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