title: Translational stem cell therapy for amyotrophic lateral sclerosis.
abstract: Effective treatments are urgently needed for amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease characterized by the loss of motor neurons. In 2009, the FDA approved the first phase I safety trial of direct intraspinal transplantation of neural stem cells into patients with ALS, which is currently in progress. Stem cell technologies represent a promising approach for treating ALS, but several issues must be addressed when translating promising experimental ALS therapies to patients. This article highlights the key research that supports the use of stem cells as a therapy for ALS, and discusses the rationale behind and approach to the phase I trial. Completion of the trial could pave the way for continued advances in stem cell therapy for ALS and other neurodegenerative diseases.
authors.affiliation: Department of Neurosurgery, Emory University, 1365-B Clifton Road NE, Atlanta, GA 30322, USA.
authors.forename: Nicholas M
authors.initials: NM
authors.lastname: Boulis
authors.forename: Thais
authors.initials: T
authors.lastname: Federici
authors.forename: Jonathan D
authors.initials: JD
authors.lastname: Glass
authors.forename: J Simon
authors.initials: JS
authors.lastname: Lunn
authors.forename: Stacey A
authors.initials: SA
authors.lastname: Sakowski
authors.forename: Eva L
authors.initials: EL
authors.lastname: Feldman
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D004195:Disease Models, Animal; D006801:Humans; D008279:Magnetic Resonance Imaging; D009414:Nerve Growth Factors / Q000494:pharmacology / Q000627:therapeutic use; D033581:Stem Cell Transplantation / Q000379:methods* / Q000639:trends
chemical_list: D009414:Nerve Growth Factors