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.

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  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.
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  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.
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  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.
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