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Monday, February 16, 2015

More Promising Data on Stem Cell Transplant for MS

Megan Brooks
February 13, 2015

Autologous hematopoietic stem cell transplant (AHSCT) proved "unequivocally" superior to mitoxantrone in reducing imaging evidence of disease activity in patients with multiple sclerosis (MS) refractory to conventional therapy in a phase 2 randomized controlled trial.
Over 4 years, patients who received AHSCT experienced 79% fewer new T2 lesions compared with patients receiving mitoxantrone. Differential treatment effects in favor of AHSCT were also seen for gadolinium-enhancing (Gd+) lesions.
The Autologous Haematopoietic Stem Cell Transplantation trial in MS (ASTIMS) study findings "strongly support phase 3 studies with primary clinical endpoints," Giovanni Mancardi, MD, from the University of Genova in Italy, and colleagues conclude.
The study was published online February 11 in Neurology.
Promoting Tolerance
The rationale for AHSCT in MS is to reset the immune system and induce a prolonged tolerance toward self-antigens.
Participants in the ASTIMS study included 21 patients with secondary progressive or relapsing-remitting MS, who had a worsening in the last year on the Expanded Disability Status Scale (EDSS) despite conventional therapy (interferon-β, glatiramer acetate, or immunosuppressive therapy), plus one or more Gd+ areas on MRI.
Nine patients were randomly allocated to intense immunosuppression (mobilization with cyclophosphamide and filgrastim, conditioning with carmustine, cytosine-arabinoside, etoposide, melphalan, and antithymocyte globulin) followed by AHSCT and 12 to 20 mg of mitoxantrone every month for 6 months.
The primary endpoint was the cumulative number of new T2 lesions in the 4 years following randomization; secondary endpoints were the cumulative number of Gd+ lesions, relapse rate, and disability progression.
Continue reading this Stem Cell therapy update

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