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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. 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 February 2018 from a small, open label, phase 1 stem cell trial at the Tisch MS Research Center of New York. This trial used 20 individuals’own mesenchymal stem cells to derive more specific stem cells called “neural progenitor cells.” The cells were expanded in the laboratory and then injected into the space around the spinal cord (intrathecal). The goal was to inhibit immune mechanisms and to augment tissue repair.

The team reported mild to moderate headache and fever as the most common side effects, and for the primary outcome of the study, which was safety, they reported no serious adverse events. Although the study was not designed to determine potential benefits of this therapy, the team reported clinical observations including some improvements in EDSS scores, strength in some muscle groups, walking speeds and bladder symptoms. Larger, longer-term, controlled studies are needed to determine the safety and effectiveness of using this approach to treat MS. 

The Tisch Center team has launched a phase II trial of this therapy. The National MS Society has agreed to partially support this trial of intrathecal stem cells in progressive MS. THIS TRIAL IS RECRUITING PARTICIPANTS. READ MORE AT CLINICALTRIALS.GOV   
• A team from Israel’s Hadassah University Hospital has been 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 phase 1/2 trial of umbilical cord-derived mesenchymal stem cells in 60 participants with MS is underway at the University of Jordan. Participants receive intrathecal injection of cells with or without the addition of physical therapy, or receive physical therapy alone. This trial is ongoing.
• A clinical trial involving IV infusion of participants’ own mesenchymal cells is taking place at the Health Sciences Centre in Winnepeg, Manitoba, and at the Ottawa Hospital in Ontario. The trial involves 40 participants with active MS. This trial is ongoing.
• A phase 1/2 clinical trial is testing IV infusion of participants’ own mesenchymal cells. The trial involves 30 people with active MS, and is taking place at three sites in Spain: University Hospital Reina Sofia in Córdoba, University Regional Hospital Carlos Haya in Málaga, and University Hospital Virgen Macarena in Sevilla. This trial is ongoing.

• A small, open-label, phase 1 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 longer, larger trials.

A phase 2 followup study has just begun. It is an 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 people with progressive MS. The study is being sponsored by BrainStorm Cell Therapeutics Inc. THIS TRIAL IS  RECRUITING PARTICIPANTS IN CALIFORNIA.
• A Phase 1 trial allogenic human neural stem cells in secondary progressive MS is underway in Italy and Switzerland. The trial is testing four different dosages injected into the brain. This trial is ongoing.
• A phase 1/2a open label study of escalating doses of intrathecal SCM-010 (adipose derived expanded mesenchymal cells) in secondary progressive MS at the Tel Aviv Medical Center in Israel. THIS TRIAL IS NOT YET RECRUITING PARTICIPANTS.

• A small, open label, phase 1 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.

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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