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New high-risk treatment can halt MS, claim researchers

pharmafile | June 10, 2016 | News story | Research and Development multiple sclerosis, research 

The results of a Phase II clinical trial, published in The Lancet, suggests that a new use of chemotherapy followed by autologous haematopoietic stem cell transplantation has fully halted the progression for multiple sclerosis (MS) for a sustained, long-term period.

23 out of 24 patients on the trial experienced no clinical relapses or development of new brain lesions while eight of these had a sustained improvement in their disability seven and a half years after treatment.

However, the researchers from University of Ottawa express a note of caution with this method as a potential treatment as this aggressive form of treatment bears the risk of serious complications associated with autologous haematopoietic stem cell transplantation (aHSCT).  

MS is one of the most common chronic inflammatory diseases of the central nervous system, affecting around two million people worldwide. While some specialist centres offer aHSCT treatment, which involves harvesting bone marrow stem cells from the patient in conjunction with chemotherapy to “reset” the immune system to stop it attacking the body, many patients relapse over time.

Instead of using chemotherapy to express the immune system, as is commonly done, the researchers destroyed the immune system completely using a chemotherapy regime of busulfan, cyclophosphamide and rabbit anti-thymocyte globulin. The primary outcome measure of the trial was MS activity-free survival at three years, which was achieved in 69.6% of patients.

Dr Mark S. Freeman, one of the lead investigators on the trial, says: “The sample size of 24 patients is very small, and no control group was used for comparison with the treatment group. Larger clinical trials will be important to confirm these results.”

Dr Emma Gray, head of clinical trials at the Multiple Sclerosis Society in the UK, comments: “This type of stem cell transplantation is a rapidly evolving area of MS research that holds a lot of promise for people with certain types of MS. This treatment does offer hope, but it’s also an aggressive procedure with substantial risks and requires specialist aftercare.”

Sean Murray

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