Stem cell transplants may work better than existing drug for severe multiple sclerosis

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Stem cell transplants may be more effective than the drug mitoxantrone for people with severe cases of multiple sclerosis, according to a new study published in Neurology, the medical journal of the American Academy of Neurology.

The study, supported by the Italian Multiple Sclerosis Foundation, involved 21 people whose disability due to multiple sclerosis had increased during the previous year even though they were undergoing first-line treatments. The participants, who were an average age of 36, were at an average disability level where a cane or crutch was needed to walk.

In this phase II study, all of the participants received medications to suppress immune system activity. Then 12 of the participants received the drug mitoxantrone, which reduces immune system activity. For the other nine participants, stem cells were harvested from their bone marrow. After the immune system was suppressed, the stem cells were reintroduced through a vein. Over time, the cells migrate to the bone marrow and produce new cells that become immune cells. The participants were followed for up to four years.

“This process appears to reset the immune system,” said study author Giovanni Mancardi, of the University of Genoa in Italy. “With these results, we can speculate that stem cell treatment may profoundly affect the course of the disease.” Intense immunosuppression followed by stem cell treatment reduced disease activity significantly more than the mitoxantrone treatment. Those who received the stem cell transplants had 80% fewer new areas of brain damage—T2 lesions—than those who received mitoxantrone, with an average of 2.5 new T2 lesions for those receiving stem cells compared to eight new T2 lesions for those receiving mitoxantrone. For gadolinium-enhancing lesions, none of the patients who received the stem cell treatment had a new lesion during the study, while 56% of those taking mitoxantrone had at least one.

Mancardi noted that the serious side effects that occurred with the stem cell treatment were expected and resolved without permanent consequences. “More research is needed with larger numbers of patients who are randomised to receive either the stem cell transplant or an approved therapy, but it is very exciting to see that this treatment may be so superior to a current treatment for people with severe multiple sclerosis that is not responding well to standard treatments,” Mancardi said.