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Multiple Sclerosis Patients Improve After Adult Stem Cell Therapy

The Lancet Neurology, January 30, 2009

Physicians at Northwestern University in Chicago have reported dramatic improvement in patients who were treated with adult bone marrow stem cells for multiple sclerosis (MS). Led by Dr. Richard Burt, the doctors treated eleven women and ten men who had had MS for approximately 5 years but who were still in the early stages of the relapsing-remitting form of the disease, and who had been unresponsive to conventional medical treatment. The adult stem cell therapy consisted of autologous hematopoietic stem cells that were extracted from each patient's own bone marrow, and follow-up monitoring of the patients was conducted for 3 years.

The adult stem cell therapy was found to halt, and in some cases even reverse, the progression of the disease. Although all of the 21 patients who participated in the clinical trial improved, 17 patients exhibited improvement that was measurable by one point or more on a standardized disability scale, which is considered to be a statistically significant improvement. MRI scans and other imaging techniques showed that remyelination had occurred, and further testing revealed that the stem cell therapy had "cleansed" the immune system of defective white blood cells.

The study confirms similar results obtained by doctors Gianluigi Mancardi and Riccardo Saccardi at the University of Genoa in Italy earlier last year.

Approximately 400,000 people in the U.S. alone have been diagnosed with various forms of MS, which is an autoimmune disease that is characterized by progressive neurological degeneration and which has previously been considered incurable.

Prior to receiving the autologous stem cell transplantation in the clinical trial led by Dr. Burt, each patient also underwent immunological myeloablation, in which radiation is employed to destroy the patient's immune system. While such a procedure has previously been considered a necessary part of the therapy, even though it exposes the patient to potentially life-threatening risks, today an increasing number of doctors are questioning the logic and necessity of subjecting their patients to deliberate immune destruction. In a publication that appeared in the Journal of Translational Medicine in January of 2007, Dr. Neil H. Riordan et al. posed the following question: "...in patients who are not suffering from a disease that is associated with an aberrant bone marrow such as hematological malignancies or immunological dysfunctions, how is it justifiable to subject them to the high levels of morbidity and mortality associated with immune suppression?" Dr. Riordan and his team of scientists then examined compelling evidence which strongly indicates that pre-transplant immune suppression is unnecessary for many types of autologous hematopoietic cell therapies and even for some allogeneic therapies that utilize "universal donor" cells such as mesenchymal stem cells and the CD34+ stem cells that are found in umbilical cord blood. As Dr. Riordan and his colleagues wrote in their 2007 paper in a section that is subtitled, "Mesenchymal stem cells do not need myeloablation for efficacy": "Currently there are several ongoing clinical trials in Phase I-III using 'universal donor' mesenchymal stem cells in non-conditioned recipients of Crohn's disease, GVHD (graft-versus-host disease) and myocardial infarction. Although these cells are bone marrow expanded mesenchymal cells, the superior proliferative potential of cord blood mesenchymal cells may allow them not only to escape immune destruction, but also to expand in vivo and mediate therapeutic effects superior to those derived from bone marrow. The fact that regulatory agencies have allowed advancement of 'off-the-shelf' universal donor mesenchymal stem cells supports the numerous reports of clinical efficacy in an allogeneic setting."

Nevertheless, as Dr. Burt and his colleagues at Northwestern University have reported, their adult stem cell procedure, even though it was preceded by the systematic destruction of each patient's immune system, "not only seems to prevent neurological progression but also appears to reverse neurological disability" in multiple sclerosis, and the results "imply that this is a valuable alternative to the transplant conditioning therapies used so far."

One can only conclude, therefore, that patients would exhibit even greater improvement if they did not have to recover from the deliberate and life-threatening destruction of their immune systems prior to receiving the stem cell therapy, and also if the stem cell therapy would utilize the "superior proliferative potential" of the "immune privileged" adult stem cells that are found in umbilical cord blood.



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