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Stem Cell Treatments for MS

A review of more than 400 treatment cases shows 60% to 70% of patients with multiple sclerosis (MS) undergoing stem cell transplants had a 3-year progression-free survival, with treatment-related morbidity of 1% to 2%. In vitro studies also indicate an end to MS causative autoimmune reactions, with clinical remission persisting after full normal immune reconstitution. Prospective randomized trials for stem cell treatment of MS were started in 2008 in Europe and the USA.

  1. What Is Multiple Sclerosis?

    • MS, also known as disseminated sclerosis or encephalomyelitis disseminata, is a chronic inflammatory and degenerative disease of the central nervous system (CNS). Autoimmunity against myelin sheaths is observed in MS and leads to demyelination of neurons. Loss of myelin and plaque formations (scleroses) in white matter of the CNS disrupts neural signaling, producing physical and cognitive impairment. The cause remains unknown.

    Stem Cells

    • Stem cells used for the treatment of MS are derived from adult bone marrow. These hematopoietic stem cells (HSCs) are multipotent in nature. HSCs are capable of self-renew and differentiation into cells of all blood lineages. Transplanted HSCs can also differentiate into specific cell types (e.g., muscle, skin, liver, lung, and neural cells).

    Treatment

    • Pharmaceutical treatments for MS focus on the inflammatory mechanisms of the disease. These treatments are of limited efficacy in suppressing immune-mediated CNS inflammation and clinical relapses. After promising studies in animal models of autoimmune diseases, immunosuppression followed by HSCs was tested as treatment in severe forms of MS.

    Benefits

    • The benefits of HSC therapy for MS include HSCs entering the brain and producing new neural cells. Remyelination has also been observed. HSC transplantation is also associated with changes in thymic output. These changes include a new and diverse T-cell receptor (TCR) repertoire.

    Results

    • HSC transplantation can promote CNS repair and regeneration via different modes of action. Proposed mechanisms include cell replacement, remyelination, and bystander activity (immunomodulation). Inflammatory activity is also reduced by treatment. Two prospective randomized trials are in progress in Europe and the USA. This studies are the first to clinically test if stem cell transplantation is superior to immunosuppressive treatments in severe cases of MS.

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