Stem Cell Transplantation in Multiple Sclerosis.

TitleStem Cell Transplantation in Multiple Sclerosis.
Publication TypeJournal Article
AuthorsCui J Y, Kraft GH, Bowen JD, McSweeney PA, Georges GE, Nash RA
JournalJournal of Spinal Cord Medicine

Objective: We are evaluating the therapeutic roles of high-dose immunosuppressive therapy (HDIT) rescued with autologous stem cell transplantation (SCT) in the management of patients with severe, nonresponsive multiple sclerosis (MS). Our hypothesis was that by ablating the immunoactive cells in a patient with MS and replacing them with nonconditional naïve cells, the disease process could be stopped. Design/Methods: We enrolled 26 patients with severe MS, including primary progressive MS (n = 7), secondary progressive MS (n = 18) and relapsing remitting MS (n = 1). Their median age was 41 years (range, 27-60). The median expanded disability status scale (EDSS) at HDIT was 7.0 (5.0-8.0). Eligibility requirements included an EDSS from 5.0 to 8.0 and deterioration of 1 or more points over the previous year. Twent-one patients had previous therapy with interferon-beta fail, and 15 had multiple therapies including Copaxone, prednisone, and methotrexate fail. The median follow-up was 12 months (range, 3-36 months). Results: With a minimum follow-up of 6 months (n = 22; 19 > 12 months), most patients remain neurologically stable with unchanged EDSS. Six patients had improvement on EDSS, 5 have had an increase in EDSS. No change was greater than 10 EDSS point. Only 2 patients had enhancing lesions on brain MRI at 1 year after SCT. One of these two had a flare of MS associated with the administration of granulocyte-colony stimulating factor. No patient has received further treatment with interferon-beta or Copaxone after SCT. Conclusion: We believe that HDIT/SCT may become an effective treatment to control severe, progressive, and refractory MS. The procedure seems to be effective. This and additional study should be continued to fully assess the long-term efficacy in these heterogeneous high-risk group patients.

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