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For decades, physicians have relied on stem cell transplants to reset the immune system of patients who have hematologic cancers, such as leukemia and lymphoma. This has led researchers to wonder if stem cell transplants could also reset the immune system so that it would not attack the patient’s own body in autoimmune diseases, such as Multiple Sclerosis, Lupus, and Rheumatoid Arthritis.
Today’s adult patients are undergoing autologous transplants with stem cells harvested from their bone marrow or peripheral blood, but today’s children could use their own cord blood for autologous transplants if they develop an autoimmune disease later in life.
One new approach to treating Multiple Sclerosis (MS) patients using Mesenchymal Stem Cells (MSC) is a phase 1 clinical trial at the Tisch MS Research Center in New York (NCT01933802). The trial, led by center director Dr. Saud A Sadiq, uses autologous MSC from the patient’s bone marrow which they manipulate in the laboratory to derive “neural progenitor” stem cells (pre-clinical publication). In this Tisch trial the patients do not receive chemotherapy, and the stem cells are administered via three intrathecal injections (injections into the cerebrospinal fluid) of 2-10 million cells per dose at 3 month intervals. Although this Tisch trial was only intended to assess the safety of the intrathecal method of stem cell delivery, they report that 70% of 10 MS patients treated so far had improvement in their level of disability.
In this article, Parents Guide to Cord Blood Foundation, has prepared two figures to illustrate the importance of cell therapy clinical trials for autoimmune diseases and the variety of cell types currently in clinical trials for these diagnoses and they are presented below for your review. The database includes 58 clinical trials for autoimmune diseases, with a total planned enrollment of 2192 patients.
Figure 1: In this pie chart, the width of the pie slices is the number of patients that the clinical trials aim to enroll, and slices are color-coded by autoimmune diagnosis. The most common diagnosis is Multiple Sclerosis at 40% of patient enrollment.
Figure 2: In this bar graph, the height of each bar is the number of patients that the clinical trials aim to enroll. There are separate bars for various types of cell sources, and within each bar they are color-coded by whether the source is autologous or allogeneic. In this graph 1256 (57%) patients are receiving autologous cell therapy and 1351 (62%) patients are receiving allogeneic Mesenchymal Stem Cell (MSC) therapy.
Many of the above clinical trials are relying on stem cells to modulate the patient’s immune system, but not to completely replace it. A worldwide review article published by Chen & Ding in Feb. 2016 identified 16 trials using MSC cell therapy for MS patients, conducted in 11 countries and using a variety of graft sources: MSC from bone marrow, from umbilical cord tissue, and from adipose tissue. Our accompanying article finds that among 58 clinical trials for any autoimmune disease that were registered over the past five years, 62% of patients are receiving MSC therapy.
The transition to treating autoimmune diseases with Mesenchymal Stem Cells MSC instead of Hematopoietic Stem Cells HSC leads to many new options for patients. Today’s children could use Mesenchymal Cells from their own cord tissue or placenta for personalized stem cell therapy of any autoimmune disease they may develop later in life.
http://parentsguidecordblood.org/en/news/cell-therapy-clinical-trials-autoimmune-diagnoses-2011-2015
LFLN REF: 03052016, p.115-116
