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Efficient Way to Generate iPSCs for the Treatment of Multiple Sclerosis

Oct 13, 2014 1:00:01 PM / by Shweta

Scientists have developed an accelerated protocol to generate functional oligodendrocytes for the treatment of multiple sclerosis

In one of our previous blogs we have described that Multiple Sclerosis (MS) is a chronic autoimmune disease of the central nervous system (CNS) characterized by brain inflammation, which arises when the nervous system is attacked by the body’s own immune cells. MS can develop because of viral infection, genetic defects or environmental factors. As yet, the exact etiology of the disease is unfortunately unknown.

Multiple Sclerosis Scientists at Columbia University, New York, have developed an accelerated protocol to generate functional oligodendrocytes to treat multiple sclerosis. Image Credit:

Currently available treatments mainly focus on targeting the immune system in order to control brain inflammation, which causes demyelination. Unfortunately, these existing treatments can only reduce or prevent the intermittent episodes of inflammation; they are not effective against the disease's progression. Furthermore, therapeutic options for patients affected with the primary progressive form (PPMS) are limited to symptomatic treatments, and the long-term prognosis is generally poor in this case.

Recently, stem cell transplantation has shown promising results to promote remyelination and induce neuro-regeneration in patients suffering from multiple sclerosis, using different sources of therapeutic cells such as human embryonic stem cells (hESCs) and induced pluripotent stem cells[1,2]. The studies in animal models of MS also have demonstrated the benefits of stem cell transplantation therapy.

The generation of patient-specific cells from induced pluripotent stem cells (iPSCs) or somatic cell nuclear transfer offers new opportunities for the treatment of many incurable neurologic diseases. Wang et al. rescued a hypomyelinated mouse model using iPSC-derived oligodendrocyte progenitor cells (OPCs) [3]. These kind of promising studies boost the hope for future clinical trials for the treatment of MS. However, oligodendrocyte differentiation protocols are still inefficient and require over 120 days in culture. Therefore, an improved protocol that can generate large numbers of purified OPCs in a relatively short time is highly desirable.

Recently, Dr. Valentina Fossati and her group at Columbia University, New York, have pioneered an efficient and robust protocol for the generation of iPSC-derived OPCs from PPMS patients [4]. Through this protocol, oligodendrocytes can be differentiated from neural stem cells to OLIG2+ progenitors and finally to O4+ OPCs in a significantly shorter time than the 120–150 days. Furthermore, PPMS-derived iPSCs were able to differentiate into MBP+ mature oligodendrocytes in vitro. In vivo myelination from PPMS-iPSC-derived OPCs, with no abnormal growths, was observed.

It has now been established that pluripotent stem cells have tremendous therapeutic potential for several diseases. If you need customized stem cells with established protocols for your research, or for cell therapy, visit HemaCare, a leading provider of stem cells.


[1.] Brustle O., et al. Embryonic stem cell-derived glial precursors: a source of myelinating trans- plants. Science 285, 754–756 (1999).

[2.] Muller F.J., et al. Gene therapy: can neural stem cells deliver? Nat. Rev. Neurosci. 7, 75–84 (2006).

[3.] Wang S., et al. Human iPSC-derived oligodendrocyte progenitor cells can myelinate and rescue a mouse model of congenital hypomyelination. Cell Stem Cell 12, 252–264 (2013).

[4.] Douvaras P, et al. Efficient Generation of Myelinating Oligodendrocytes from Primary Progressive Multiple Sclerosis Patients by Induced Pluripotent Stem Cells. Stem Cell Reports, Vol. 3: 250–259 (August 2014)

Topics: disease-state cells, iPSCs, Regenerative Medicine, Stem Cell Therapy, Stem Cells, tissue engineering, Basic Research


Written by Shweta

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