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Multiple sclerosis and stem cells

The original article in this thread summarises ways in which different stem cell therapies are being tested for multiple sclerosis (MS), particularly in animal models. The research in this area is particularly driven by the need to develop therapies for aggressive MS that has proven unresponsive to other existing therapies. It is considered to be a promising means of intervention in such cases, but caution is needed as there are some safety concerns.

Autologous haematopoietic stem cell transplantation has been tried in various countries, including the Czech Republic, Italy and China in both single-centre and multi-centre trials. In Italy, patients were treated with an intermediate intensity conditioning regimen, named BEAM/ATG. Observations on patients treated between the years 1996-2008. Study of outcomes for these patients suggested that the regimen helped suppress or slow disease progression and caused sustained clinical improvement in patients who were unresponsive to conventional therapies. The Czech experience with patients with aggressive, unresponsive MS at a single centre was similar. Both countries reported that the stem cell treatment was especially beneficial in patients in the relapsing-remitting phase of the disease. A retrospective analysis of outcomes in 25 MS patients in a single centre in China similarly reported that autologous haematopoietic stem cell transplantation is a feasible treatment for severe MS with good long-term efficacy. Studies on phenotype and function of peripheral blood lymphocytes from MS patients undergoing a trial of myeloablative autologous haematopoietic stem cell transplantation suggests that the transplantation favourably modifies the balance of regulatory and pro-inflammatory lymphocytes to allow suppression of central nervous system inflammation.

However, caution is necessary when deciding whether or not to use autologous haematopoietic stem cell transplantation. Some of the studies mentioned reported major early toxicity and deaths directly due to the transplants. A case study from Costa Rica of a 17-eyar old girl who underwent stem cell transplantation nine months after being diagnosed with MS reported that she suffered life-threatening encephalomyelitis as a result of the therapy, although she eventually recovered. The therapy is still experimental at this stage and more development with formal, controlled clinical trials is necessary before it could be more generally introduced as a therapy for MS. Such trials are currently underway.

Sources

ALDERAZI, Y.J., COONS, S.W. and CHAPMAN, K., 2012. Catastrophic demyelinating encephalomyelitis after intrathecal and intravenous stem cell transplantation in a patient with multiple sclerosis. Journal of child neurology, 27(5), pp. 632-635

CHEN, B. et al., 2012. Long-term efficacy of autologous haematopoietic stem cell transplantation in multiple sclerosis at a single institution in China. Neurological Sciences: Official Journal Of The Italian Neurological Society And Of The Italian Society Of Clinical Neurophysiology, 33(4), pp. 881-886

KRASULOVÁ, E. et al., 2010. High-dose immunoablation with autologous haematopoietic stem cell transplantation in aggressive multiple sclerosis: a single centre 10-year experience. Multiple sclerosis (Houndmills, Basingstoke, England), 16(6), pp. 685-693

MANCARDI, G.L. et al., 2012. Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen in multiple sclerosis: the Italian multi-centre experience. Multiple sclerosis (Houndmills, Basingstoke, England), 18(6), pp. 835-842

PATANI, R. and CHANDRAN, S., 2012. Experimental and therapeutic opportunities for stem cells in multiple sclerosis. International Journal Of Molecular Sciences, 13(11), pp. 14470-14491

SACCARDI, R. et al., 2012. A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Multiple sclerosis (Houndmills, Basingstoke, England), 18(6), pp. 825-834