Stem Cells Show Protective Potential for Amyotrophic Lateral Sclerosis (ALS)

Stem Cells Show Protective Potential for Amyotrophic Lateral Sclerosis (ALS)

Stem cells are being increasingly used to create therapies to address diseases across a number of organs. Neurodegenerative disease is one category for which there has been an abundance of research into the potential of stem cells to slow symptom progression or reverse symptoms altogether. For some neurodegenerative diseases, such as Parkinson’s disease, the approach for using stem cells is to create a therapy that enables the cells that are lost through the disease to be replaced by new cells. It is the hope of scientists and clinicians that this type cell replacement therapy will halt aspects of the disease that result from cell loss.

Recently, scientists published experimental results in PLoS One that demonstrate the potential of another type of stem cell approach. More specifically, these researchers showed that in the case of amyotrophic lateral sclerosis (ALS), using stem cells to help create a protective environment for existing cells may have a positive impact on disease progression. The specific type of stem cells used in this experiment was the mesenchymal stem cell.

While cell replacement therapies often employ mesenchymal stem cells because of their ability to differentiate into many different cell types, the researchers in this study used them because of their promise for enhancing the health of a cell environment. Indeed, these cells are known to have immunomodulatory properties and to fight inflammation.

The researchers specifically looked at how mesenchymal stem cells could affect motor neurons and glia because these are the cell types that are implicated in ALS. What they found was that these stem cells were able to reduce apoptosis, a process whereby cells self-destruct as a result of cues in the environment that alert the cells to unhealthy conditions. Critically, the extent to which this type of cell destruction was minimized depended on the amount of mesenchymal stem cell that was present. In other words, with more stem cells came more protection.

Another important observation was that mesenchymal stem cells were associated with the expression of important growth factors that are known to support healthy environments and to reduce markers of inflammation, which tend to be associated with unhealthy environments.

Together, these results suggest that ALS could potentially be addressed with stem cells without the development of aggressive cell replacement therapies. Instead, stem cells could be used to create protective environments for neurons that are normally affected by the disease.

Stem Cells that Secrete Neurotrophic Factors Shown to be Safe in ALS Patients

Stem Cells that Secrete Neurotrophic Factors Shown to be Safe in ALS Patients

Amyotrophic lateral sclerosis, known as ALS, is only partially understood, and there is no effective prevention or treatment of the disease. Those who are diagnosed with ALS, therefore, have few options for slowing its progression. The disease attacks cells of the nervous system called motor neurons. Thus, while one option for approaching ALS is to prevent the attack of motor neurons, another approach is to find a way to protect those cells of the nervous system.

Small proteins called neurotrophic factors have previously been shown to help motor neurons grow and survive longer. However, when scientists have tried to apply these neurotrophic factors in ALS patients, they have not made a difference in disease progression. Because stem cells offer a way to replace damaged cells, they are studied as potential candidates for therapeutic interventions in neurodegenerative diseases, such as ALS.

In this study, scientists combined stem cells and neurotrophic factors by inducing mesenchymal stem cells to secrete neurotrophic factors. Their main objective was to determine if injecting this type of stem cell into ALS patients is safe. They also aimed to determine if such cells may be clinically helpful for this group of patients.

The scientists gave 6 patients each the stem cells through two different types of injections. They then gave 14 patients a combination of the two types of injections. All patients were between the ages of 20 and 75 and were in the early stages of ALS. The patients were observed for 6 months following their injections. Any side effects that occurred as a result of the stem cell injections were mild or transient, so each of the techniques for administering the stem cells to ALS patients was deemed safe.

The researchers also used a test called the ALS-Functional Rating Scale-Revised to evaluate the clinical impact of the stem cell treatment on ALS patients. They found that 13 of the patients (or 87%) improved in their performance on the test, suggesting that the stem cells may have improved ALS symptoms. Further research will need to be conducted to determine if these mesenchymal stem cells that secrete neurotrophic factors can indeed help ALS patients – and if so, how they can best be used to combat the disease.

Phase I Clinical Trial Demonstrates Safety of Stem Cell Therapy for Amyotrophic Lateral Sclerosis

Phase I Clinical Trial Demonstrates Safety of Stem Cell Therapy for Amyotrophic Lateral Sclerosis

A recent publication in Stem Cells Translational Medicine described an open-label phase I clinical trial that was designed to assess the safety of a stem cell therapy for amyotrophic lateral sclerosis (ALS). The specific procedure involved two repeated injections of autologous bone marrow derived mesenchymal stem cells into the spinal canal of patients with ALS. The researchers found that the injections were safe during their implementation and that they did not have any unsafe effects during a follow-up period of one year.

The phase I clinical trial included 8 patients whose ALS was either definite or probable. The patients underwent a procedure to have mesenchymal stem cells isolated from their bone marrow, and the stem cells were expanded outside the patients’ bodies for 28 days. Unfortunately, a patient died before treatment, so only 7 of the patients then had the spinal canal injections of the stem cells. The two injections were given 26 days apart.

Conventional tests to evaluate the status of ALS in patients were used to assess the impact of the stem cells on the patients’ disease. In the 6 months following the injections, the disease progression did not accelerate, according to the ALS Functional Rating Scale-Revised (ALSFRS-R) score. There were also no serious adverse events observed over a 12 month follow up. Some adverse side effects occurred but subsided with or without treatment within a few days.

The significance of this study is the observation that this particular stem cell therapy procedure appears safe for ALS patients. Further research will help likely determine how stem cell therapy for amyotorophic lateral sclerosis can be used to slow the disease progression, while maintaining high degrees of safety.

Learn more about stem cell therapy for ALS here.

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