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FDA APPROVES STEM CELL CLINICAL TRIAL

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    FDA APPROVES STEM CELL CLINICAL TRIAL

    FDA approves stem cell clinical trial For multiple sclerosis
    (14/08/13)
    The Tisch MS Research Center of New York announced today that it has received Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) to commence a Phase 1 trial using autologous neural stem cells in the treatment of multiple sclerosis (MS). MS is a chronic human autoimmune disease of the central nervous system that leads to myelin damage and neurodegeneration and affects approximately 2.5 million people worldwide.

    "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, Senior Research Scientist at Tisch MS Research Center of New York and the study's principal investigator.

    The groundbreaking study will investigate a regenerative strategy using stem cells harvested from the patient's own bone marrow. These stem cells will be injected intrathecally (into the cerebrospinal fluid surrounding the spinal cord) in 20 participants who meet the inclusion criteria for the trial. This will be an open label safety and tolerability study. All study activities will be conducted at the Tisch MS Research Center and affiliated International Multiple Sclerosis Management Practice (IMSMP).

    The clinical application of autologous neural progenitors in MS is the culmination of a decade of stem cell research conducted by a dedicated team of scientists headed by Dr. Sadiq and by Dr. Violaine Harris, Research Scientist at Tisch MS Research Center.

    Preclinical testing found that the injection of these cells may decrease brain inflammation and promote myelin repair and/or neuroprotection. "This study exemplifies the Tisch MS Research Center's dedication to translational research and provides a hope that established disability may be reversed in MS," Dr. Sadiq noted.

    Participants will undergo a single bone marrow collection procedure, from which mesenchymal stem cell-derived neural progenitor cells (MSC-NPs) will be isolated, expanded and tested prior to injection. Participants will receive three rounds of injections at three month intervals. Safety and efficacy parameters will be evaluated in all participants through regular follow-up visits.

    ABOUT TISCH MS RESEARCH CENTER OF NEW YORK For over twenty years, Dr. Saud A. Sadiq has believed that combining excellence in clinical care with innovative research targeted at finding the cure for multiple sclerosis would set an exemplary standard in the treatment of MS patients. Today, the Tisch MS Research Center of New York embodies this new model of healthcare, in which your doctor is also your researcher. Dr. Sadiq helps those with MS by conducting cutting-edge, patient-based research to ensure unparalleled care. The close relationship of the non-profit research center and its affiliated clinical practice (International Multiple Sclerosis Management Practice) enables the testing of new MS treatments and accelerates the pace at which research discoveries move from lab bench to bedside. The Tisch MS Research Center of New York aims to identify the disease trigger, optimize treatments for patients, and repair the damage caused by multiple sclerosis.

    Source: Digital Journal copyright © 2013 digitaljournal.com (14/08/13)

    #2
    Further from Tisch MS Research site. 20 PROGRESSIVE PATIENTS to be enrolled

    FDA Approves MSC-NP Therapy as Investigational New Drug in MS Clinical Trial: A Research Milestone

    The stem cell research division of the Tisch MS Research Cener of New York proudly announces Food and Drug Administration (FDA) approval of autologous, mesenchymal stem cell-derived neural progenitor cells (MSC-NPs) as an Investigational New Drug (IND) for an open label, phase I clinical trial in the treatment of multiple sclerosis. Approximately 20 progressive MS patients, recruited from the existing patient population of the International Multiple Sclerosis Management Practice (IMSMP), will be initially enrolled. The Tisch MS Research Center stem cell trial is the first of its kind in the United States, and incorporates the following key advantages:

    MSC-NPs derived from adult autologous mesenchymal stem cells are the primary therapeutic agent.
    MSC-NPs display a greater potential for differentiation into mature neural tissue, with lower risk for ectopic differentiation.
    MSC-NPs display a greater potential for differentiation into mature neural tissue, with lower risk for ectopic differentiation.
    MSC-NPs have the capacity for recruitment of existing stem cells within the brain and spinal cord via the induction of immunomodulatory and trophic growth factors.
    MSC-NPs will be administered in multiple rounds of treatment rather than a single treatment.
    Route of administration will be intrathecal (into the cerebrospinal fluid) in order to directly target regenerative mechanisms in the central nervous system.
    This FDA approval is the culmination of more than a decade of research into the therapeutic potential of stem cells for MS patients and confirmation of the pioneering approach adopted by the Tisch MS stem cell research team led by Saud Sadiq, MD and Violaine Harris, PhD. The proof-of-concept of this approach was demonstrated in the EAE animal model of MS (Harris et al., J Neurol Sci, 2012). This study showed compelling evidence of the therapeutic potential of intrathecal MSC-NPs in mice with clinically established EAE, which modeled their potential benefit in patients with progressive MS. Pathological findings from the EAE study demonstrated that injected MSC-NPs migrated to areas of demyelination where they seemed to influence the rate of repair through effects on endogenous progenitors in the spinal cord. Furthermore, this study informed the clinical trial design in terms of dosing and dosing frequency. The clinical translation of this stem cell research was further advanced in a publication examining the characteristics of human autologous MSC-NPs from patients with MS (Harris et al., Stem Cells Translational Medicine, 2012). This study showed that bone marrow derived MSC-NPs from MS patients were a feasible source of stem cells for clinical application. Lastly, in an ongoing pilot clinical study, we continue to monitor seven MS patients who received intrathecal autologous MSC-NP infusions between 2005 and 2007 in support of the long-term safety of MSC-NP therapy for MS (Harris et al., manuscript in preparation).

    In related stem cell work, we have shown that the immunoregulatory and trophic properties of mesenchymal stem cells may enhance engraftment and remyelinating capability of co-transplanted oligodendrocyte progenitor cells (Cristofanilli et al., Stem Cells and Development, 2011). More recently, we have explored the capability of the CNS milieu, and specifically cerebrospinal fluid from patients with MS, to support repair through stimulation of an endogenous pool of stem cell (Cristofanilli et al., Neuroscience, 2013).

    As important a milestone as the FDA-IND approval is, it marks the true beginning and not the end of our clinical research of stem cell therapy. In this and subsequent studies, we hope to define the optimal therapeutic dose and dosing frequency of stem cells, the best route (or combined routes) of administration, whether or not stem cells should be used in combination with other disease modifying treatments, and which patients are most likely to benefit from this treatment.

    The primary objective of the Phase I clinical trial will be to determine safety of autologous MSC-NPs in MS patients who meet inclusion/exclusion criteria after baseline screening. Intrathecal injections of isolated and expanded autologous MSC-NP cells will be performed over a six month period. Study participants will have follow-up visits at multiple timepoints during the treatment phase of the study, and up to 27 months after the final injection. The secondary objective of the trial is to observe trends in efficacy over the course of the three year study.

    Enrollment in the clinical trial will commence after approval of the protocol has been obtained from an Institutional Review Board (IRB) and funding has been secured. A definitive date for enrollment to begin will be announced at that time, prior to which no study activities will be conducted. Questions about the study protocol, enrollment criteria, and other non-clinical matters should be directed to the Study Coordinators Daniel Koffler (dkoffler@tischms.org or 646-557-3852) and Sydney Chirls (schirls@tischms.org or 646-557-3860). Clinical questions will be forwarded to one of the research nurses.

    Comment


      #3
      Originally posted by AMJ View Post
      Preclinical testing found that the injection of these cells may decrease brain inflammation and promote myelin repair and/or neuroprotection. "This study exemplifies the Tisch MS Research Center's dedication to translational research and provides a hope that established disability may be reversed in MS," Dr. Sadiq noted.
      May this study prove to be the answer we're all looking for!
      Kimba

      “When you change the way you look at things, the things you look at change.” ― Max Planck

      Comment


        #4
        'From your lips...' Good luck

        Comment


          #5
          Great catch, AMJ. I looked up this story in about 3 posts on the internet, mostly all the same, but it looks like a good thing to me.
          I am from Philadelphia and I would drive to the Tisch center, in a heartbeat, if there was any chance that I could be a study participant.

          Comment


            #6
            If the FDA has approved this clinical trial, is there a fee to the patient? The participants are going to be recruited from the existing patient population of the International Multiple Sclerosis Management Practice (IMSMP). Is this part of the Tisch Research Centre as the article says they are affiliated?

            I am happy that they are looking to enroll progressive patients. This is good news. My hope is for stem cell treatment to become available and affordable for everyone.

            Comment


              #7
              Dr. Burt is conducting another HSCT trial at Northwestern and , like all the other trials he has done in the last few years, progressive forms are 'excluded'. So he continues to perform the same protocol, on the same types of patients, to keep his clinics' 'batting avg.' up. All the while, ignoring about 15% of the MS population.... Such is life with MS. Sorry...Just a rant.
              Maybe, if there is a God, the Tisch trial will turn into something for all of us. Good luck

              Comment


                #8
                I saw Dr. Sadiq 15 years ago. He did a treatment for my MS that was controversial. I am convinced thats why I have been symptom free all these years, however I did have an attack recently so it's time maybe to give him a call again and fly out to NY.

                To be honest the idea of all that chemo with dr. burt I was not crazy about.
                Suspected MS 1985. dx 1994 still RRMS EDSS 1.0

                Comment


                  #9
                  Both studies provide some hope.

                  Comment


                    #10
                    Anyone know when they start recruiting?
                    Suspected MS 1985. dx 1994 still RRMS EDSS 1.0

                    Comment


                      #11
                      I am watching very closely. As you are, I suppose !

                      Comment


                        #12
                        is this the same thing as the the other treatment dr. burt is doing? Is this supposed to stop progression or is this to hopefully repair past damage.
                        Suspected MS 1985. dx 1994 still RRMS EDSS 1.0

                        Comment


                          #13
                          The article that I read stated that this study is to investigate the 'regenerative' possibilities of using stem cells infused into the spinal cord. This is not, IMHO, what happens in Dr. Burt's clinic. Additionally, Dr. Burt's protocol involves chemotherapy and this hasn't been stated in any reports that I have read about the trial at Tisch center. If you find out any information on this study, let us in on it. Good luck

                          Comment


                            #14
                            I think Dr. Burts is to stop progression and maybe Dr. Sadiqs is to regenerate? So both are different. I was hoping Dr. Sadiqs was to stop the MS process as well.
                            Suspected MS 1985. dx 1994 still RRMS EDSS 1.0

                            Comment


                              #15
                              I just found out that the clinical trial that was approved to be rub by the Tisch MS Research Center will get their participants from patients that are being treated by their own 4 neurologists, Dr. Sadiq, included. The practice is called the International Multiple Sclerosis..... something or other. It seems that it also going to take about 2 years to complete and the participants will need to be in New York, for treatment, a lot of times. And being a patient of that practice doesn't 'guarantee' inclusion as a study participant.

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