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Mesenchymal Stem Cells and iPSCs


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Mesenchymal Stem Cells and iPSCs

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” and “induced pluripotent 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.

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

Adult mesenchymal stem cells are being tested in clinical trials in MS. The approach is similar to HSCT, except that the individual’s immune cells are not destroyed or replaced. Instead, 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. 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:
• A small, open label, phase I stem cell trial is nearly completed at the Tisch MS Research Center of New York using individuals’own mesenchymal stem cells to derive more specific stem cells called “neural progenitor cells.” The cells are expanded in the laboratory and then injected into the space around the spinal cord (intrathecal). The goal is to inhibit immune mechanisms and to augment tissue repair. The trial was based on the team's previous small pilot study, results of which were published in October 2016. The pilot study, involving 6 people with progressive MS, tested the safety, feasibility and tolerability of infusing different doses of mesenchymal stem cells. The team reported that the procedure appeared to be safe after an average followup of 7 years. THE PHASE I TRIAL IS NEARING COMPLETION AND NOT SEEKING ADDITIONAL PARTICIPANTS. 
Recently the Tisch Center team announced intentions of following-up on this work by conducting a phase II trial of this therapy. The National MS Society is currently in discussions with the team to determine how the proposed phase II trial may be funded in an expedited fashion, pending successful completion of the phase I trial, and that its results continue to show that the procedure is safe. 
• An international, placebo-controlled, phase II stem cell trial involving people with MS who show specific signs of inflammation and active disease, including people with primary progressive MS, secondary-progressive MS and relapsing-remitting MS, is getting underway at multiple sites in Europe and Canada.  The “MESEMS” trial will test benefits and safety of using individuals’ own bone marrow cells, which are extracted and then given by intravenous infusion immediately or six months after extraction. The goal is to inhibit immune mechanisms and to augment tissue repair. THIS TRIAL IS BEGINNING TO RECRUIT PARTICIPANTS. GO TO THE MESEMS WEBSITE FOR FURTHER INFORMATION ABOUT LOCATION AND CONTACT INFORMATION.  

• A team from Israel’s Hadassah University Hospital is conducting a 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 (EDSS scale 3.5-6.5). They are administering 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. As of a report given at a medical conference (ECTRIMS) in September 2016, of 39 enrolled people, they have not found serious adverse events, with the most common side effect being headache. This trial is ongoing

• A small, open label, phase I trial of stem cells derived from placenta (known as “PDA-001” manufactured by Celgene Cellular Therapeutics) was completed in 2014, and results suggested this approach was safe. The study involved 16 people with relapsing-remitting or secondary-progressive MS at sites in the U.S. and Canada. This study was designed to evaluate safety and not designed to show effectiveness. In the published paper, the researchers comment that the next step, a proof-of-concept clinical trial, is planned. THIS PLANNED NEXT TRIAL IS NOT YET RECRUITING PARTICIPANTS.

• A placebo-controlled, phase II stem cell trial  involving people with secondary-progressive MS and primary progressive MS has begun at Frenchay Hospital in Bristol, United Kingdom, testing the benefits and safety of using individuals’ own bone marrow cells. The cells are extracted and then given by intravenous infusion immediately or one year after the extraction. The goal is to inhibit immune mechanisms and to augment tissue repair. THIS TRIAL IS RECRUITING PARTICIPANTS AT ONE SITE IN THE UNITED KINGDOM. 

• A small, open-label, phase I clinical trial at Cleveland Clinic tested the ability of an individual’s own mesenchymal stem cells to both inhibit immune mechanisms and to augment intrinsic tissue repair processes in people with relapsing forms of MS. They were given intravenously (infused into the vein). This study was led by Dr. Jeffrey A. Cohen and supported by the Congressionally Directed Medical Research Programs. The National MS Society provided support for a pilot study related to this trial to compare stem cells from people with MS and controls without MS, looking at how the cells survive and function, to enhance understanding from this stem cell trial. This trial, which was designed to evaluate safety and not designed to determine benefits, was completed and preliminary results were published in April 2017, suggesting that this approach was safe and warrants a phase 2 trial, which is now in planning stages. THIS PLANNED TRIAL IS NOT YET RECRUITING ADDITIONAL PARTICIPANTS.

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.

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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