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National MS Society Research in Stem Cells

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The National MS Society is the largest private funder of MS research in the world and is recognized as a global leader in driving breakthroughs to a cure. The Society stimulates studies worldwide, leverages opportunities, fosters collaboration among foremost experts, and shapes the research landscape to address the urgent needs of people with MS. 

The complexity of MS necessitates a comprehensive approach to address our most pressing research priorities and to accelerate research breakthroughs. Our current multi-year commitments total over $60 million to drive solutions that will assist every single person with MS to live their best lives. To make the most progress for everyone, we pursue all promising paths. One of these paths is stem cells, including adult stem cells that act as “spare parts” inside the body.

Repairing myelin, the coating that surrounds and protects axons (nerve wires) and which is damaged by MS, may represent the best strategy for protecting axons from injury and improving function for people with MS. Thanks in part to our pioneering funding, potential cell therapies and myelin repair strategies are now approaching or already in clinical trials.

There is exciting progress being made through innovative research related to the potential of many types of stem cells both for slowing MS disease activity and for repairing damage to the nervous system. In addition to cell therapy, finding ways to stimulate the body’s own stem cells inside the brain is another approach being actively investigated. Today the idea of nervous system repair holds significant promise as a strategy to restore the function that MS has taken from people; and reducing or stopping MS progression. 

We are at a pivotal moment in time where breakthrough solutions can change the world for everyone with MS. Among the approaches being taken are:
  • Clinical trials of individuals’ own blood or bone marrow stem cells to “reboot” the immune system to slow or halt MS activity.
  • Research to slow disease activity and repair nervous system damage directly with stem cells that may replace the cells that make myelin.
  • Research and clinical trials to stimulate the natural capacity of the brain to repair itself. 
This remarkable progress is due in large part to the National MS Society’s comprehensive efforts and multi-million dollar research investments. With the urgent need for more effective treatments for MS, particularly for those with more progressive forms of the disease, we believe that the potential of all types of cell therapies and other ways to help restore function must be explored.

The Society is supporting research projects exploring various types of stem cells, including cells derived from bone marrow, fat and skin, and has supported 68 stem cell studies over the past 10 years. In addition, a substantial portion of the Society’s research portfolio focuses on different approaches to protecting and repairing the nervous system. 

Leadership Role

The Society has been at the forefront to drive progress and consensus in this promising field.
  • In 2005, the National MS Society convened a Task Force on Stem Cell Research. Among its findings, the task force recommended that the Society hold a scientific symposium in early 2007 involving leading stem cell experts from around the world to further explore the viability of all types of stem cell research for the treatment, prevention and cure of MS.
  • This stem cell summit was held on January 16-19, 2007 in San Francisco, California. Over 75 experts in the areas of stem cells and MS participated in the Summit. A scholarly review of this meeting along with recommendations on priorities for moving this important research forward were published in a medical journal. 
  • A May 20, 2009 Stem Cell meeting organized by the MS Society in the UK and USA, and supported by the MS Society of Canada, Italy, France, Australia and the MS International Federation, was a response to this urgent need for guidance to the MS research community. The result was international consensus on the future of stem cell transplantation research for people with MS, paving the way for more coordinated global research efforts and potentially better, and quicker, patient access to stem cell clinical trials.
  • With research rapidly advancing, in November 2015, the International Conference on Cell-Based Therapy for Multiple Sclerosis was convened in Lisbon, Portugal under the auspices of the International Advisory Committee on Clinical Trials in MS (a group jointly sponsored by the National MS Society and the European Committee for Treatment and Research in Multiple Sclerosis).
    • The Conference was chaired by Jeffrey A. Cohen, MD (Cleveland Clinic, USA), Marcelo C. Pasquini, MD, MS (Medical College of Wisconsin, USA) and Neil Scolding, PhD, FRCP (Southmead Hospital Bristol, UK).  
    • Participants reviewed current experience with, and value of, specific cell-based therapies. Invited speakers Drs. Saud Sadiq (Tisch MS Research Center of New York) and Richard Burt (Northwestern University) joined 70 other leading researchers and clinicians who conferred on clinical trials needed to provide answers about which types of cells, which route of delivery, and which types and stages of disease, would be the most promising approach for treating MS.
    • A review of the findings and consensus on next steps has been published by the conference organizers, with recommendations to help speed the development of new cell-based treatment solutions.  Read about this review
  • The Society has been engaged with the team planning a phase 3 trial of HSCT and is encouraging quick action to design and launch the trial. We are working with the research team to explore ways to get this moving more quickly.
  • The Society convened a working group of the National Medical Advisory Committee, other stem cell researchers and people with MS to advise the Society on the information and resource needs for healthcare professionals and people living with MS. Two short webinars were developed to better define stem cells and how they might be used in MS. National MS Society website content was updated and an FAQ section was added to provide guidance on the dangers of unregulated stem cell clinics who claim cures for MS and other neurodegenerative conditions.    

Recent Projects Supported by the National MS Society

To help drive breakthroughs to a cure, the National MS Society has supported research for more than a decade into the potential of different types of stem cells, including cells derived from bone marrow, fat and skin. See Glossary for definitions of terms used here.
  • The Tisch MS Research Center of New York is conducting a phase II clinical trial to see whether individuals' own bone marrow-derived mesenchymal stem cells can inhibit immune mechanisms and augment tissue repair. From those cells, more specific “neural progenitor cells” are derived. The cells are expanded in the laboratory and then injected into the space around the spinal cord (intrathecal). The National MS Society has agreed to partially support this trial, which involves participants with progressive MS.
  • Investigators are the University of Connecticut Health Center are exploring the therapeutic potential of stem cells and a novel method of tracking the course of secondary progressive MS in mice.
  • University of Utah researchers testing the idea that molecules secreted by stem cells improve potential for repairing nerve-insulating myelin.
  • Albert Einstein College of Medicine researchers optimizing ways of producing myelin-making cells to speed efforts to find strategies to repair nerve-insulating myelin and restore function in MS.
  • Researchers at The State University of New York at Buffalo are attempting a new strategy to improve the ability of cells to repair of nerve-insulating myelin.
  • A research grant at the University of Glasgow, Scotland, focusing on whether adult stem cells from the nose can dampen harmful aspects of the immune system and improve myelin repair in rodent models of myelin loss.
  • Investigators in Milan, Italy received a research grant to do preclinical testing of adult stem cells (iPSC) to stimulate repair of damaged myelin.
  • Researchers at Johns Hopkins University studied the ability of different types of transplanted stem cells to modulate the immune system and promote repair in MS models.
  • A Collaborative Center MS Research Center at the University of California, Irvine, focused on the potential of stem cells for treating MS.
  • Investigators in Paris, France used myelin-making cells from outside the brain and spinal cord (peripheral nervous system) in attempts to repair MS damage in MS models.
  • University of California, Davis researchers investigated the potential of using cells derived from adult skin to repair nerve-insulating myelin damaged during the course of MS.
  • Researchers at the University of Rochester Medical Center studied myelin repair cells in action in mice for clues to stimulating nervous system repair in MS.
  • Investigators at Yale University evaluated the transplantation of myelin-producing cells to repair damaged myelin in an animal model, for clues to the possible safety and benefit in people with MS.
  • Cleveland Clinic investigators studied the best way to track mesenchymal stem cells in the body during clinical trials of this novel strategy for treating MS.
  • Stanford University scientists devised methods to use skin cells to produce myelinating cells in sufficient quantities for transplantation in MS models as a prelude to their possible use in people with MS.
  • As part of a Collaborative MS Research Center at the Mayo Clinic, researchers are taking a novel approach to studying nerve cells and possible ways to protect them. This includes creating stem cells by reprogramming skin cells from people with MS, growing them in lab dishes to create “MS in a dish,” enabling close study of any abnormalities and the ability to test therapies to fix the problem.
At present, there are no approved stem cell therapies for MS. 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.

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