Hematopoietic Stem Cell Transplantation: MS Breakthrough …

Multiple Sclerosis (MS) is a devastating disease for many who become afflicted with the diseases progressive form, often in the prime of their lives, with no cure and when the effectiveness of established MS treatment is so often disappointing. Discouragement can lead to pinning premature hope on unproven therapies that seem to hold promise.

For example, withchronic cerebrospinal venous insufficiency (CCSVI or CCVI) the so-called liberation therapy developed by Italian researcher Paolo Zamboni in 2008, Dr. Zamboni hypothesized that compromised flow of blood in the veins draining the central nervous system played a role as the cause or in the development of multiple sclerosis, and devised a procedure dubbed liberation therapy by the media that involves angioplasty (stenting) of key veins in an attempt to improve blood flow.

CCSVI was and still is greeted with skepticism within the medical community, and Wikipedias entry on the topic notes that Dr. Zambonis first published research was neither blinded nor did it have a comparison group, and that Dr. Zamboni also did not disclose his financial ties to Esaote, manufacturer of the ultrasound specifically used in CCSVI diagnosis.

The so-called liberation procedure has also been criticized for possibly resulting in serious complications and deaths while its benefits have not been proven, with the United States Food and Drug Administrations position being that it is not clear if CCSVI exists as a clinical entity, and that these treatments may cause more harm. Wikipedia notes that CCSVI research has been fast-tracked, but researchers thus far still have been unable to confirm whether CCSVI has a role in causing MS, consequently raising serious objection to the hypothesis of CCSVI originating multiple sclerosis. Research continues, and a 2013 study found that CCSVI is equally rare in people with and without MS, while narrowing of the cervical veins is equally common.

This writer is personally aware of specific cases in which CCSVI has seemed to result in substantial improvement in MS symptoms, and others where it proved ineffective. The jury, as they say, is still out, but a cautious approach seems to be prudent.

Likewise for another controversial new therapy being advocated for MS and other autoimmune diseases, known as high-dose immunosuppressive therapy with hematopoietic stem cell transplantation in MS and other autoimmune diseases, in which clinicians destroy the patients immune system with chemotherapy, and then reboot it with stem cells. Dr. Denis Federenko of the A.A. Maximov Hematology and Cell Therapy Department of the National Pirogov Medical Surgical Centre in Russia specializes in this treatment, pointing out that conventional therapies do not provide satisfactory control of multiple sclerosis; hormonal therapy helps to limit acute manifestations of the disease, but doesnt stop its progression; and Interferon therapy may help some patients, but in most cases it does not provide a stable long-term effect.

The Russian proponents of this approach to treating MS contend that chemotherapy eliminates the cause of the disease the autoimmune T-cells that are responsible for nerve tissue damage. Then the patient is transplanted with his/her own (autologous) stem cells, which were collected and frozen in advance. They say that over the past decade more than 700 patients have received this treatment, which they claim may stop progression of the disease in most patients and prevent further deterioration of their quality of life, and that MS patients will not need any maintenance therapy after transplantation. Claimed efficiency of high-dose immunosuppressive therapy with hematopoietic stem cell transplantation in multiple sclerosis approximates 75%-80%, and is most effective in young patients with rapidly progressing multiple sclerosis in its early stages, when the leading mechanism of the damage to the nervous system is autoimmune inflammation. In later stages of the disease, after irreversible damage is done, they caution that transplantations effect is limited.

In an opinion statement entitled Autologous hematopoietic stem cell transplantation as a treatment option for aggressive multiple sclerosis (Curr Treat Options Neurol. 2013 Jun;15(3):270-80. doi: 10.1007/s11940-013-0234-9) published in the journal Current Treatment Options in Neurology, N. Pfender, R. Saccardi, and R. Martin of the Department of Neurology at University Hospital Zurich in Zurich, Switzerland, observe that Despite the development of several injectable or oral treatments for relapsing-remitting multiple sclerosis (RRMS), it remains difficult to treat patients with aggressive disease, and many of these continue to develop severe disability.

The authors observe that over the last two decades, hematopoietic stem cell transplantation (aHSCT or HSCT) has been explored, and clinical studies have shown that aHSCT is able to completely halt disease activity in the majority of patients with aggressive RRMS, and that research on the mechanisms of action supports that aHSCT indeed leads to renewal of a healthy immune system.

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Hematopoietic Stem Cell Transplantation: MS Breakthrough ...

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