A multicenter, phase II trial from Genova, Italy has been completed on 21 patients with severe multiple sclerosis (MS), published in March, 2015.
These were patients whose MS was not under control despite conventional MS treatments. They developed new brain lesions that were documented on MRI scans. In the first phase of the trial all participants received intense immunosuppressant therapy to suppress the autoimmune cells of the immune system. In the second phase 12 patients received mitoxantrone, a standard chemotherapeutic agent used conventionally for complicated MS cases.
The other 9 patients had bone marrow samples removed. When the research team determined that the immune system was fully suppressed, they received their own bone marrow transplant to restore their hematopoietic cells in the bone marrow.
As the next step of the study both groups of patients were observed for 4 years. At the end MRI scans of the brain of all participants were taken. The stem cell group had 79% fewer brain lesion than the mitoxantrone group. On average there were 2.5 brain lesions per patient in the stem cell group over 4 years while the control group on mitoxantrone developed 8 lesions on average.
However, the multidisciplinary researchers analyzed other parameters of disease severity. The main study author, Dr. Giovanni Mancardi said: “This process appears to reset the immune system.” And he added: “With these results, we can speculate that stem cell treatment may profoundly affect the course of the disease.”
There were serious side effects with the stem cell group. Unfortunately details of these side effects were not given. But each case could be worked out, and no long-term effect from the side effects remained. Dr. Mancardi mentioned that the adverse events were anticipated, but the clinicians were able resolve them without permanent consequences.
All patients of the trial were using canes and crutches to cope with their disabilities since an average age of 36 years prior to entering the trial. There was no difference with regard to the progression of disability between the two groups. Whatever neurological lesion had been causing disability likely was due to scarring that would not be expected to resolve with stem cell treatment.
Comment: At this point this is an interesting new approach to severe forms of MS that are extremely difficult to treat. However, we need to wait for the completion of a phase III trial with more patients before this can be recommended as a standard therapy. Also, one wonders whether the bone marrow, which presently is given intravenously should not be partially given intrathecally (into the space where the cerebrospinal fluid envelops the spinal cord and the brain). This approach may suppress brain lesions and spinal lesions even more. The stem cell literature seems to indicate to me that stem cells need to be brought as closely as possible to the area that is problematical. This is not a therapy that should be taken lightly! The stem cell treatment is not the problem, but the pre-treatment chemotherapy can have serious consequences.
More info on treatment of multiple sclerosis: https://nethealthbook.com/neurology-neurological-disease/multiple-sclerosis/treatment-multiple-sclerosis/