A new approach to using stem cells for multiple sclerosis (MS) treatment has been tried at Northwestern University of Chicago. Dr. Richard Burton and colleagues treated the worst form of MS. This is called relapsing-remitting multiple sclerosis. It is known to lead to early disabilities. 145 patients were treated between 2003 and 2014. The final follow-up was done in June 2014. A bone marrow aspirate was taken from the patients initially. This served as a source for the later bone marrow transplant. The patients then received cyclophosphamide and alemtuzumab. Alternatively they received cyclophosphamide and thymoglobulin. This was intended to weaken the autoimmune reactions in the body causing MS. Subsequently the bone marrow stem cells were given intravenously.
Assessing success of stem cells for multiple sclerosis
Various scores were used to assess patients’ progress. The Expanded Disability Status Scale (EDSS) score assessed the disability status. The Neurologic Rating Scale (NRS) determined the neurological status. The Multiple Sclerosis Functional Composite (MSFC) and a “quality of life” scale assessed general functioning. They also assessed the number of MS lesions on MRI scans of the head. At the 4-year mark the progression-free survival was 87%, which is impressive given that they were treating relapsing-remitting multiple sclerosis. After 2 years the quality of life score had improved from 46 pre-bone marrow transplant to 64. There was a decrease in MS lesions in the brain, measured as volume. Before the bone marrow transplant the volume of the average was 8.57 cubic centimeters. 27 months following the bone marrow transplant the lesion had shrunk on average to 5.74 cubic centimeters. This was measured in 128 patients.
Similar favorable results were also reported from another group, this time in New York: Dr. Sadiq is specializing in treating young people with MS at the Tisch MS Research Center. He has developed a method where bone marrow stem cells are modified in tissue culture into brain-like stem cells, called neural stem cells. These are subsequently injected into the spinal fluid space through lumbar puncture. The hope is that the insulation material of the nerves that has broken down in MS patients is being produced again. With his first 10 patients he had a response rate of 70% (7 out of 10). This has never been possible in the past.
Conclusion of using stem cells for multiple sclerosis
New approaches have been introduced to treat MS. MS is a chronic neurodegenerative condition that leads to premature disabilities. In the past a complete obliteration of the bone marrow was thought to be necessary. However, this led to a high complication rate from the treatment. Now a much lower chemotherapy dosage is used to treat patients. Subsequently the bone marrow transplant is given. With this method a response rate of progression-free survival of 87% was achieved. In younger patients it has been shown that MS can be treated with no chemotherapeutic pretreatment. In this case bone marrow stem cells were modified into neural stem cells. A 70% response rate to this therapy was achieved.
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