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FDA Approves Unique Stem Cell Therapy Trial in MS Patients

FDA Approves Unique Stem Cell Therapy Trial in MS Patients

Stem cell research continues to excite the field of MS research as a new Phase I trial involving the use of stems cells as an Investigational New Drug (IND) in MS patients has been approved by the Food and Drug Administration (FDA).  The small Phase I clinical trial will investigate the safety and efficacy of autologous, mesenchymal stems cell-derived neural progenitor cells (MSC-NPs) in the treatment of progressive MS.

Neural progenitor cells are cells that can develop into neurons and other nervous system cells, such as oligodendrocytes, encouraging neural repair and regeneration as evidenced in studies with mice (Harris, 2012).  Mesenchymal stem cells (MSCs) are adult stem cells derived from bone marrow which can differentiate into other types of cells such as neural progenitor cells (NPCs).  Autologous mesenchymal stem cells are obtained from a person’s (or animal’s) own bone marrow.

Led by Saud Sadiq, MD and Violaine Harris, PhD, researchers at the Tisch MS Research Center, formerly known as the Multiple Sclerosis Research Center of New York (MSRCNY), will be recruiting approximately 20 progressive MS patients from the existing patient population of the International Multiple Sclerosis Management Practice (IMSMP), which is the research center’s clinical practice affiliate in Manhattan, to participate in this open label, clinical trial.  Enrollment has not yet begun.

In this trial, the stem cells will be taken from each patient’s own bone marrow from which MSC-NPs will be isolated, expanded and tested prior to injection.  Patients will then receive three rounds of intrathecal (into the cerebrospinal fluid) injections of the MSC-NPs at three month intervals.  Patients will be followed for up to 27 months following the final injection.  Although similar studies have been conducted in the United Kingdom, this is the first of its kind to take place in the United States.

“To my knowledge, this is the first FDA-approved stem cell trial in the United States to investigate direct injection of stem cells into the cerebrospinal fluid of MS patients, and represents an exciting advance in MS research and treatment,” said Dr. Saud A. Sadiq, the study’s principal investigator, in a statement issued by Tisch MS Research Center of New York.

Researchers at Tisch MS Research Center are excited to move forward with this clinical trial which represents the culmination of over 10 years of stem cell research.  Although the study was initially approved by the International Cellular Medicine Society (ICMS), which is an Institutional Review Board (IRB), in November 2011, it had been delayed due to regulatory issues.  Last September, the FDA advised Tisch “that a clinical trial of stem cell therapy falls under FDA jurisdiction as an Investigational New Drug (IND), and that therefore IRB approval in the absence of additional FDA-IND approval is not a sufficient legal basis for initiating the stem cell trial.” All work on the stem cell trial was suspended at that time until the regulatory issues were resolved and the FDA granted this approval.

One thing which makes this research different than previous stem cell research conducted in the United States is that it does not involve administration of high-dose chemotherapeutic drugs to drastically lower the patient’s immune system before stem cell transplantation as in the ongoing HALT-MS (High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis) Study conducted by NIH.  This trial also involves multiple intrathecal, rather than intravenous, injections of stem cells to directly target regenerative mechanisms in the central nervous system.

View references below

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This article represents the opinions, thoughts, and experiences of the author; none of this content has been paid for by any advertiser. The MultipleSclerosis.net team does not recommend or endorse any products or treatments discussed herein. Learn more about how we maintain editorial integrity here.

  1. FDA Approves Stem Cell Clinical Trial for Multiple Sclerosis.  Tisch MS Research Center Press Release, August 14, 2013.  Accessed August 27, 2013 at http://www.imsmp.org/sites/default/files/news/FDA Approves Stem Cell trial press release.pdf
  2. FDA Approved MSC-NP Therapy as Investigational New Drug in MS Clinical Trial: A Research Milestone.  Tisch MS Research Center News Release.  Accessed August 27, 2013 at http://www.tischms.org/news/fda-approves-msc-np-therapy-investigational-new-drug-ms-clinical-trial-research-milestone
  3. A Statement on Stem Cell Trial Developments: September 13, 2012.  Tisch MS Research Center News Release.  Accessed August 28, 2013 at http://www.tischms.org/news/statement-stem-cell-trial-developments-september-13-2012
  4. A Statement on Stem Cell Trial Developments: August 24, 2012.  Tisch MS Research Center News Release.  Accessed August 28, 2013 at http://www.tischms.org/news/statement-stem-cell-trial-developments-august-24-2012
  5. High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) Study.  Immune Tolerance Network.  Accessed August 28, 2013 at http://www.immunetolerance.org/studies/high-dose-immunosuppression-and-autologous-transplantation-multiple-sclerosis-halt-ms
  6. Cristofanilli M, Cymring B, Lu A, Rosenthal H, Sadiq SA.  Cerebrospinal fluid derived from progressive multiple sclerosis patients promotes neuronal and oligodendroglial differentiation of human neural precursor cell in vitro.  Neuroscience. 2013 Jul 19;250C:614-621. doi: 10.1016/j.neuroscience.2013.07.022. [Epub ahead of print]
  7. Cristofanilli M, Harris VK, Zigelbaum A, Goossens AM, Lu A, Rosenthal H, Sadiq SA.  Mesenchymal Stem Cells Enhance the Engraftment and Myelinating Ability of Allogeneic Oligodendrocyte Progenitors in Dysmyelinated Mice.  Stem Cells Dev. 2011 Dec;20(12):2065-76. doi: 10.1089/scd.2010.0547. Epub 2011 Mar 12.
  8. Harris VK, Faroqui R, Vyshkina T, Sadiq SA.  Characterization of Autologous Mesenchymal Stem Cell-Derived Neural Progenitors as a Feasible Source of Stem Cells for Central Nervous System Applications in Multiple Sclerosis.  Stem Cells Transl Med. 2012 July; 1(7): 536–547. doi: 10.5966/sctm.2012-0015. Epub 2012 June 28.
  9. Harris VK, Yan QJ, Vyshkina T, Sahabi S, Liu X, Sadiq SA.  Clinical and Pathological Effects of Intrathecal Injection of Mesenchymal Stem Cell-Derived Neural Progenitors in an Experimental Model of Multiple Sclerosis.  J Neurol Sci. 2012 Feb 15;313(1-2):167-77. doi: 10.1016/j.jns.2011.08.036. Epub 2011 Oct 1.
  10. Holloman JP, Ho CC, Hukki A, Huntley JL, Gallicano GI.  The development of hematopoietic and mesenchymal stem cell transplantation as an effective treatment for multiple sclerosis.  Am J Stem Cell. 2013;2(2):95-107. ISSN:2160-4150/AJSC1305001. Epub 2013 June 30.
  11. Karussis D, Karageorgiou C, Vaknin-Dembinsky A, et al. Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis. Arch Neurol. 2010 Oct;67(10):1187-94.
  12. Mohyeddin Bonab M, Yazdanbakhsh S, Lotfi J, et al. Does mesenchymal stem cell therapy help multiple sclerosis patients? Report of a pilot study. Iran J Immunol. 2007 Mar;4(1):50-7.
  13. Tang Y, Cui YC, Wang XJ, et al. Neural progenitor cells derived from adult bone marrow mesenchymal stem cells promote neuronal regeneration.  Life Sci. 2012 Nov 2;91(19-20):951-8. doi: 10.1016/j.lfs.2012.09.005. Epub 2012 Sep 19.
  14. Yamout B, Hourani R, Salti H, et al. Bone marrow mesenchymal stem cell transplantation in patients with multiple sclerosis: a pilot study. J Neuroimmunol. 2010 Oct 8;227(1-2):185-9.

Comments

  • Candice
    6 years ago

    No, I’m just too old. I have dealt with this many years and I’m tired of these headaches I’m tired of this dizziness pain and this bed. I’m ready to go

  • Dave87
    6 years ago

    Apparently they’re picky about who they choose…I have SPMS and talked to a Mr Koffler about the trials and nothing came of it….but then again I’m not Ozzy Osbournes’ son or Richard Cohen.

  • MARLA POLLOT
    6 years ago

    SAD, ANGRY, DEPRESSED THAT MY 13 YEAR OLD SON IS GETTING SO BIG. 12 YEARS TO DIAGNOSE. NOT MUCH POSITIVE TO SAY EXCEPT BRING ON THE STEM CELL TRANSFUSION. I WOULD DO IT IN A HEARTBEAT.

  • Lisa Emrich moderator author
    6 years ago

    Marla,
    I’m sorry to read that you are having a hard time. Research into stem cell therapy really is exciting. I hope that scientists are able to discover the most effective methods of helping to heal damage from MS and/or stopping the disease in its tracks. That would be cool.
    Lisa

  • MARLA POLLOT
    6 years ago

    E MY EYES, WHICH ARE NOT VERY GOOD EITHER.

  • Maris
    6 years ago

    Does anyone know if anything is happening with this in Israel?

  • Lisa Emrich moderator author
    6 years ago

    Hi Maris,
    I’m not aware of any specific trials going on in Israel. Searching clinicaltrials.gov may reveal studies which are taking place in Israel. Unfortunately, a search of “multiple sclerosis and Israel” also turns up studies taking place at Beth Israel Deaconess hospital in Boston, Massachusetts (USA).
    Lisa

  • Drkdreamer
    6 years ago

    Is this trial a duplicate of one already done in Europe?

    I begin to think sharing results of medical trials between countries is as difficult as the CIA sharing with the FBI and NSA.

  • Lisa Emrich moderator author
    6 years ago

    Hi Drkdreamer,

    I agree that sharing results of clinical research between institutes and across borders would be helpful to speed research. This is one of the ideas behind the research work supported by the Myelin Repair Foundation.

    A study was published last Nov (2012) that involved very similar research to the research study mentioned above. However, it only involved one injection of mesenchymal stem cells into the cerebrospinal fluid (rather than 3) and patients were following for 12 months (instead of two years). see http://www.ncbi.nlm.nih.gov/pubmed/23061813

    Another study from Cambridge administered the stem cells intravenously (rather than into the spinal fluid). see http://www.ncbi.nlm.nih.gov/pubmed/22236384

    So, it’s not quite a duplicate, but is very similar.

    Lisa

  • Dalene
    6 years ago

    Where are these trials being done? I was in the Optic Neuritis Treatment Trials in the late 80’s and into the 90’s. I would be interested in participating.

  • Lisa Emrich moderator author
    6 years ago

    Hi Dalene,

    The Tisch MS Research Center is located in New York. According to their website, enrollment will not begin until they receive new IRB approval. (see http://www.tischms.org/news/fda-approves-msc-np-therapy-investigational-new-drug-ms-clinical-trial-research-milestone)

    Contact information for questions regarding the trial is provided at the bottom of the page listed above.

    Lisa

  • PegLeg
    6 years ago

    Hi,, I am secondary progressive,,and I took a leap of faith,, and did the Stem Cell Genex,, results,, nada,, it started to work, but it quit,,so I hope everyone who tries, has good results,, I won’t try it again,,

  • Lisa Emrich moderator author
    6 years ago

    Hi PegLeg,

    Thanks for your comment. I’m sorry to hear that the treatment you underwent didn’t ultimately work for you. There is so much research going on with different types of therapies that hopefully nerve regeneration will be predictable.

    Lisa

  • Karen Martinez
    6 years ago

    Where can I read about the trials in the United Kingdom that have already been done? I am excited to hear that the FDA finally approved the trials..they only set us back one year. I guess nobody at the FDA has MS.

  • Lisa Emrich moderator author
    6 years ago

    Hi Karen,

    You can search clinicaltrials.gov to find completed, ongoing, and some future trials using stem cells in MS (http://www.clinicaltrials.gov/ct2/results?term=multiple+sclerosis+stem+cell). The search results can be further divided into the country of origin. That’s where I happened to notice trials occurring in the UK, as well as other European countries.

    If you like looking through abstracts, you can also search through abstracts, posters, and some webcasts conducted at last year’s 2012 ECTRIMS conference. I didn’t look specifically for stem cell trials, but there is a lot of interesting material included.

    Lisa

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