Tisch MS Research Center Launches Indiegogo Campaign to Raise $300,000 for Stem Cell Trial
Last week more than 300 MS Activists visited their elected officials on Capitol Hill to ask for increased funding for research at the National Institute of Health (NIH) and the Congressionally Directed Medical Research Program (CDMRP) which have been instrumental in conducting vital research into the causes, treatments, and understanding of multiple sclerosis. Frequently, small biotech companies and large pharmaceutical companies conduct the clinical research of which we are most aware and that ultimately leads to approved therapies. We are less aware of the vital early stage research which forms the foundation of larger clinical trials. This research is often supported through government funding and (MS) society funding which some industry funding.
The Tisch MS Research Center of New York, formerly known as the Multiple Sclerosis Research Center of New York (MSRCNY), is a non-profit research organization which is conducting vital research into the use of stem cells to promote remyelination to repair the damage caused by MS. Much discussion in the MS community is that remyelinating agents and stem cell therapies are where the most exciting developments will be found in the treatment of MS in the future.
In August 2013, the Tisch MS Research Center, led by Saud Sadiq, MD and Violaine Harris, PhD, received approval by the Food and Drug Administration (FDA) to conduct a new Phase I trial involving the use of stems cells as an Investigational New Drug (IND). 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.
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.
Researchers at Tisch MSRCNY are excited to move forward with this clinical trial which represents the culmination of over 10 years of stem cell research. However, this first round of research involving 20 patients is expected to cost $600,000 and can’t be completed without money. Tisch is an independent research center which is funded through charitable donations.
So, today, Tisch launched a crowd-funding campaign on Indiegogo to raise an initial $300,000 by April 14, 2014 to begin the study. At the time of posting, they have already raised $104,980 towards their goal. With Indiegogo, any donation amount is accepted and Tisch MSRCNY will receive the funds even if their goal is not met by April 14. But the MS community can help Tisch meet and exceed this goal simply by spreading the word.
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Tisch has provided information regarding their research center and a basic explanation of using stem cells to promote repair in MS on their Indiegogo campaign page. For more background information regarding stem cell research in MS and the Tisch MSRCNY, please check the references listed below.
SOURCES and RELATED REFERENCES
FDA approves unique stem cell therapy trial in MS patients. Lisa Emrich, MultipleSclerosis.net, August 28, 2013.
Tisch MS Center’s FDA-Approved Phase I Stem Cell Trial for Multiple Sclerosis. Indiegogo Campaign, launched March 17, 2014.
FDA Approves Stem Cell Clinical Trial for Multiple Sclerosis. Tisch MS Research Center Press Release, August 14, 2013.
FDA Approved MSC-NP Therapy as Investigational New Drug in MS Clinical Trial: A Research Milestone. Tisch MS Research Center News Release, August 14, 2013.
A Statement on Stem Cell Trial Developments: September 13, 2012. Tisch MS Research Center News Release.
A Statement on Stem Cell Trial Developments: August 24, 2012. Tisch MS Research Center News Release.
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]
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.
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.
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.
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.
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.
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.
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.
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.