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Small, Uncontrolled Study Looks at Long-Term Effects of Bone Marrow Stem Cell Transplantation for MS

March 24, 2011

In a small uncontrolled study, 35 people with MS underwent bone marrow stem cell transplantation aimed at “rebooting” the immune system, and were followed for two to 15 years. Two deaths occurred which were attributed to the treatment, and three other patients died over the follow-up period, two of which were attributed to complications of their MS. The treatment showed the most benefit in people considered to have aggressive disease. This procedure is experimental, and is the subject of ongoing clinical trials to determine its benefits and risks in people with MS. Drs. A. Fassas, Vasilios K. Kimiskidis, and colleagues (Aristotle University of Thessaloniki Medical School, Greece) report their findings in Neurology (March 22, 2011 76:1066-1070).

Bone marrow transplantation is a lifesaving treatment for certain cancers. It is variously called “hematopoietic stem cell therapy” and “autologous stem cell transplantation.” People are given infusions of their own bone marrow stem cells, which are first extracted and treated. Chemotherapy and sometimes whole-body radiation are used to wipe out the person’s immune system before the bone marrow cells are reintroduced by injection. The hope is that the new immune cells will no longer attack myelin or other brain tissue, so that the person develops a new more tolerant immune system.

This procedure is strictly investigational. In clinical trials over the years, it has produced some good results in MS, usually for younger, less disabled people, however, others have seen their MS return, and experienced more progression. And, sadly, a few have died. The National MS Society’s list of Clinical Trials in MS 2010 lists three ongoing studies in the U.S. and Canada using this approach; read more about these trials.

The team from Greece conducted 35 transplants from June 1995 until May 2001 in people whose MS symptoms had progressed in the year preceding treatment. Two people died from transplant-related complications at two months and 2.5 years after the procedure, and three others died over the follow-up period, two of which were attributed to complications of their MS. Sixteen people improved on the EDSS scale measuring disability, with improvements lasting for a median of 2 years. Two of the individuals stabilized and remained improved over 7 and 8 years, respectively. Seven worsened during follow-up but remained better than their disability level at baseline, while seven others worsened compared to their disability level at baseline. The results appeared to be most beneficial in those who had evidence of active areas of disease activity (“gadolinium-enhancing lesions”) on MRI scans at the time of the transplant.

The most impressive results in this series were observed in very active “aggressive” MS cases, but whether bone marrow transplantation is superior to conventional treatments cannot be answered on the basis of this this study, note the authors, because of the methods used. This was an uncontrolled and unblinded study, meaning that no other treatment or sham procedure was used for comparison, and all participants and clinicians knew what therapy was being administered (blinding is used to control bias). Furthermore, the duration of follow-up varied widely, and many individuals who initially improved following treatment worsened to varying degrees during follow-up. The treatment-related deaths, which occurred in 6% of the study population, are of concern, and warrant caution in pursuing such experimental therapy outside of a clinical trial setting.

Read more about treatments for progressive MS.

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system. Symptoms range from numbness and tingling to blindness and paralysis, and there is currently no cure for MS. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. An estimated 1 million people live with MS in the United States. Most people with MS are diagnosed between the ages of 20 and 50, and it affects women three times more than men.


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