A study published in the journal Neurology reveals that stem cell transplantation could be a more effective therapy in severe cases of multiple sclerosis (MS) than the drug mitoxantrone. The study is entitled “Autologous hematopoietic stem cell transplantation in multiple sclerosis.”
MS is an immune-mediated disorder in which the body’s own immune system attacks the central nervous system (brain and spinal cord nerves). Hematopoietic stem cell transplantation is routinely used in the treatment of leukemia and lymphoma, and is currently being studied as a possible therapy for severe autoimmune disorders as MS.
Researchers designed a randomized phase II clinical trial study including 21 MS patients (average age of 36) whose disability due to the disease had worsened in the previous year despite the fact that the patients were under conventional medication treatment. The average disability level of the participants was represented by the need of a crutch or cane to walk. The goal of the study was to determine the efficacy of intense immunosuppression followed by either autologous hematopoietic stem cells transplantation or mitoxantrone (MTX) in MS disease activity.
All participants received immune-suppressive medication. An immunosupressive drug (MTX) was then given to 12 of the patients while the remaining 9 received hematopoietic stem cells harvested from their bone marrow. After immunosuppression, the stem cells were intravenously reintroduced in their donors and migrated back to the bone marrow where they generated new immune cells. All participants were followed-up for a period of up to four years.
“This process appears to reset the immune system,” said the lead study author Dr. Giovanni Mancardi in a news release. “With these results, we can speculate that stem cell treatment may profoundly affect the course of the disease.”
Researchers found that a robust immunosuppression approach followed by stem cell treatment resulted in a significantly higher decrease in disease progression in comparison with MTX treatment. MS patients under stem cell treatment reduced the number of new areas of brain damage (T2 lesions) by 79% compared to patients under MTX treatment. Another type of lesion seen in MS patients – gadolinium-enhancing lesions – were not detected in patients under stem cell treatment during the study, whereas 56% of patients receiving MTX exhibited at least one new lesion.The researchers concluded that an intense immunosuppression followed by autologous hematopoietic stem cell transplantation is more efficient than MTX to reduce MS activity in severe cases.
“More research is needed with larger numbers of patients who are randomized to receive either the stem cell transplant or an approved therapy, but it’s very exciting to see that this treatment may be so superior to a current treatment for people with severe MS that is not responding well to standard treatments,” concluded study author Dr. Mancardi.
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