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Autologous stem cell transplantation as rescue therapy in malignant forms of multiple sclerosis

Department of Neurosciences, Ophthalmology and Genetics, University of Genoa, Genoa, Italy, Centre of Excellence for Biomedical Research, University of Genoa, Genoa, Italy, glmancardi{at}neurologia.unige.it

Alessandra Murialdo

Department of Neurosciences, Ophthalmology and Genetics, University of Genoa, Genoa, Italy

Paolo Rossi

Department of Neurology and Clinical Neurophysiology, University Vita-Salute San Raffaele, Milan, Italy

Francesca Gualandi

II Division of Haematology and Stem Cell Transplantation Centre, S. Martino Hospital, Genoa, Italy

Gianvito Martino

Department of Neurology and Clinical Neurophysiology, University Vita-Salute San Raffaele, Milan, Italy

Alberto Marmont

II Division of Haematology and Stem Cell Transplantation Centre, S. Martino Hospital, Genoa, Italy

Fabio Ciceri

Department of Haematology, University Vita-Salute San Raffaele, Milan, Italy

Angelo Schenone

Department of Neurosciences, Ophthalmology and Genetics, University of Genoa, Genoa, Italy, Centre of Excellence for Biomedical Research, University of Genoa, Genoa, Italy

Roberto Carlo Parodi

Division of Neuroradiology, San Martino Hospital, Genoa, Italy

Elisabetta Capello

Department of Neurosciences, Ophthalmology and Genetics, University of Genoa, Genoa, Italy

Giancarlo Comi

Department of Neurology and Clinical Neurophysiology, University Vita-Salute San Raffaele, Milan, Italy

Antonio Uccelli

Department of Neurosciences, Ophthalmology and Genetics, University of Genoa, Genoa, Italy, Centre of Excellence for Biomedical Research, University of Genoa, Genoa, Italy

Malignant forms of multiple sclerosis (MS) represent a limited group of very aggressive demyelinating diseases, which rapidly progress to severe disability leading often to life-threatening conditions. On these clinical entities, currently available therapies for MS are not very effective. Recently, it has been demonstrated that intense immunosuppression followed by autologous stem cell transplantation (ASCT) can affect the clinical course of individuals with severe MS and completely abrogate the inflammatory activity detected by magnetic resonance imaging (MRI). We report on the treatment with intense immune ablation followed by ASCT of three patients with malignant MS whose clinical course indicated a dramatically poor prognosis. This procedure succeeded in halting the rapidly worsening course of disease. The effect was long lasting, as demonstrated by a sustained efficacy over a two-year period in two subjects and 12 months in the third case. In addition, a striking effect on inflammation-related MRI findings was obtained. These results support a role for intense immunosuppression followed by ASCT as treatment in rapidly evolving malignant MS cases unresponsive to conventional therapies.

Key Words: autologous stem cells transplantation • immunosuppression • MS

Multiple Sclerosis, Vol. 11, No. 3, 367-371 (2005)
DOI: 10.1191/1352458505ms1181cr


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