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Mesenchymal Stem Cells, iPSCs, and NSCs

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Mesenchymal Stem Cells, iPSCs, and NSCs

Many types of stem cells are being explored for their potential benefits for treating multiple sclerosis. This page focuses on research related to “mesenchymal stem cells,” “induced pluripotent stem cells,” and "neural stem cells" in MS. Read about other types of stem cells. 
  • “Mesenchymal stem cells” are adult stem cells found in several places in the body, including the bone marrow, skin and fat tissue.
  • “Induced pluripotent stem cells” or iPSC. These are adult stem cells such as skin cells which are “induced” or programmed in the laboratory to become stem cells.
  • “Neural stem cells” are specialized stem cells that can give rise to many types of cells in the nervous system. These can be derived from other types of stem cells.

Research on Mesenchymal Stem Cells and Neural Stem Cells to Reduce MS Disease Activity and Augment Repair

Adult mesenchymal stem cells are being tested in clinical trials in MS. A person’s own mesenchymal stem cells are isolated from the bone marrow or blood stream and multiplied in the lab, and then re-introduced in greater numbers into their body. Alternatively, the cells are sometimes treated prior to transfer to potentially enhance their ability to suppress nervous system-damaging immune responses and/or promote myelin repair.

Only when the results of clinical trials are available will it be possible to determine what the optimal cells, delivery methods, safety and actual effectiveness of this experimental approach might be for people with MS.
 
Mesenchymal stem cells are being tested in several clinical trials including:

• Results were published in 2023 by an international team that tested a novel strategy to transplant neural stem cells directly into the brains of 15 people with secondary progressive multiple sclerosis. No serious adverse events were reported, and participants’ physical and cognitive functions remained stable for one year afterward. Further study is necessary to determine the potential of this strategy for stopping MS progression and restoring function in people with MS. 

Results were presented in 2023 by Dr. Saud Sadiq (The Tisch MS Research Center of New York) from a phase 2 clinical trial involving 54 people with progressive MS, funded in part by the National MS Society. The study used participants’ own bone marrow stem cells, which were removed and expanded in the laboratory and developed into specific cells called Mesenchymal Stem Cells-Neural Progenitors. They were then infused into the spinal fluid every two months for one year. The primary purpose of the trial was to see improvement in physical mobility after the first year, which was not achieved. The stem cell treatments were well tolerated. Other outcomes may point to potential benefits of this treatment, such as improved walking, especially in those with more mobility challenges. Further study is needed to determine if this form of stem cell therapy is safe and beneficial as a treatment for progressive MS. 

Results were published in 2022 from A Phase 1 trial allogenic human neural stem cells in secondary progressive MS in Italy and Switzerland. The trial tested four different dosages injected into the brain. Treatment was feasible, tolerated and safe and participants remained clinically  stable during the 12-month follow-up. No serious adverse events occured related to the treatment. 

Results were reported in 2021 from a phase 2  open-label study of repeated intrathecal administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors, MSC-NTF) at Cleveland Clinic and Stanford University in 18 people with progressive MS. The study is being sponsored by BrainStorm Cell Therapeutics Inc. The cells were derived from individuals’ own bone marrow (mesenchymal) stem cells, which were treated in the laboratory to encourage them to secrete neuroprotective factors. The participants then received three separate cell injections into the spinal canal every two months. The treatment appeared to be safe, and there also appeared to be some improvements in cognition, vision, mobility, and other functions by comparing participants with those involved in other studies. Read the ECTRIMS abstract

Results were published in 2021 from a Hadassah University Hospital phase 2 clinical trial to establish the optimal way to administer mesenchymal stem cells in active, progressive MS. Participants are people with progressive MS with moderate to more severe disability. They administered the cells into the vein or the spinal fluid and then monitoring patients with various measures to try to detect a benefit and to find the best route for administering these cells. Most participants stabilized, and some showed improvement. Read more

•  Results were published in 2021 from the international MESEMS trial, a phase 2 clinical trial conducted at 15 sites in 9 countries, testing the ability of mesenchymal stem cells to reduce new inflammation seen on MRI brain scans of people with relapsing MS, 24 weeks after one dose of participants’ own cells were infused into their veins. The procedure was found to be safe, but it did not reduce the risk of active brain lesions, which was the main endpoint established for this trial. Read more 

Larger, longer-term, controlled studies are needed to determine the safety and effectiveness of using stem cells to treat MS. When the results of these and subsequent clinical trials are available, it should be possible to determine what the optimal cells, delivery methods, safety and actual effectiveness of these current experimental therapies might be for different people with MS. 
 
Read more about stem cell clinics.
Learn about ongoing MSC trials on clinicaltrials.gov

Research on Induced Pluripotent Stem Cells (iPSCs) for Nervous System Repair and Disease Modeling

Another line of stem cell research in MS relates to efforts to repair nervous system damage directly with stem cells that may replace the cells that make myelin, the protective cover on nerve wires which is damaged during MS, and nerve cells that have been destroyed. One exciting avenue being explored in early stages is the concept of taking samples of a person’s skin cells or other cells and turning them into stem cells. These cells are called “induced pluripotent stem cells” or iPSC. The potential advantage of this approach is that it’s possible such cells would not be rejected by the person’s immune system, and this approach bypasses possible ethical concerns connected with human embryonic stem cells.

Research using iPSCs is still in its infancy as studies proceed to determine whether any types of stem cells can reverse MS damage and restore function. Read more about efforts to repair the nervous system.

Researchers are also beginning to explore the usefulness of iPSCs for furthering our understanding of MS disease processes. In this approach, stem cells are created by reprogramming skin or other types of cells from people with MS. These are then grown in lab dishes to create “MS in a dish,” enabling close study of possible abnormalities in various types of cells. This approach also holds potential for testing possible therapies.

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