title: Stem cell treatment in Amyotrophic Lateral Sclerosis.
abstract: Amyotrophic Lateral Sclerosis is a progressive fatal neurodegenerative disease that targets motor neurons. Its origin is unknown but a main role of reactive astrogliosis and microglia activation in the pathogenesis has been recently demonstrated. Surrounding neurons with healthy adjoining cells completely stops motor neuron death in some cases. Hence stem cell transplantation might represent a promising therapeutic strategy. In this study MSCs were isolated from bone marrow of 9 patients with definite ALS. Growth kinetics, immunophenotype, telomere length and karyotype were evaluated during in vitro expansion. No significant differences between donors or patients were observed. The patients received intraspinal injections of autologous MSCs at the thoracic level and monitored for 4 years. No significant acute or late side effects were evidenced. No modification of the spinal cord volume or other signs of abnormal cell proliferation were observed. Four patients show a significant slowing down of the linear decline of the forced vital capacity and of the ALS-FRS score. Our results seem to demonstrate that MSCs represent a good chance for stem cell cell-based therapy in ALS and that intraspinal injection of MSCs is safe also in the long term. A new phase 1 study is carried out to verify these data in a larger number of patients.
authors.affiliation: Department of Neurology, Azienda Ospedaliera, Eastern Piedmont University of Novara, Novara, Italy. mazzini.l@libero.it
authors.forename: Letizia
authors.initials: L
authors.lastname: Mazzini
authors.forename: Katia
authors.initials: K
authors.lastname: Mareschi
authors.forename: Ivana
authors.initials: I
authors.lastname: Ferrero
authors.forename: Elena
authors.initials: E
authors.lastname: Vassallo
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Oliveri
authors.forename: Nicola
authors.initials: N
authors.lastname: Nasuelli
authors.forename: Gaia Donata
authors.initials: GD
authors.lastname: Oggioni
authors.forename: Lucia
authors.initials: L
authors.lastname: Testa
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
mesh_terms: D000328:Adult; D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000601:surgery*; D001854:Bone Marrow Cells / Q000502:physiology; D002454:Cell Differentiation / Q000502:physiology; D049109:Cell Proliferation; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods*; D059630:Mesenchymal Stem Cells / Q000502:physiology*; D008875:Middle Aged; D009046:Motor Neurons; D009474:Neurons / Q000502:physiology*; D013997:Time Factors; D014182:Transplantation, Autologous / Q000379:methods