title: Regenerative medicine in Huntington's disease: current status on fetal grafts and prospects for the use of pluripotent stem cell.
abstract: Huntington's disease is currently incurable, but cell therapy is seen as a promising alternative treatment. We analyze the safety and efficacy of the intrastriatal transplantation of human fetal neuroblasts from ganglionic eminences in patients with Huntington's disease. A few rare surgical incidents were reported, but the main difficulty associated with this therapeutic approach is the occurrence of recipient alloimmunization against the graft and the lack of availability, standardization and quality control for the fetus-derived products required for cell therapy. Some patients showed sustained cognitive improvement over periods of more than six years, and motor improvements for more than four years. Grafting outcomes are variable even within individual transplantation centers. The reasons for this variability are poorly understood, highlighting the need for further research in this specific area. With the perspective of additional trials in the future, we review here the development of human pluripotent stem cell-derived cell therapy products for HD, and their advantages and disadvantages with respect to fetal cells.
authors.affiliation: Unité de neurologie cognitive, centre de référence maladie de Huntington, hôpital Henri-Mondor-Albert-Chenevier, Assistance publique-Hôpitaux de Paris, 54, avenue du Général-de-Lattre-de-Tassigny, 94010 Créteil, France; Inserm U955, équipe 01 neuropsychologie interventionnelle, Institut Mondor de recherche biomédicale, 94000 Créteil, France; Faculté de médecine, université Paris Est, 94000 Créteil, France; École normale supérieure, institut d'études cognitives, 75005 Paris, France; Cognitive, hôpital Henri-Mondor-Albert-Chenevier, 94000 Créteil, France. Electronic address: bachoud@gmail.com.
authors.forename: A-C
authors.initials: AC
authors.lastname: Bachoud-Lévi
authors.affiliation: Inserm U861, I-STEM, AFM, 91030 Évry cedex, France; UEVE U861, I-STEM, AFM, 91030 Évry cedex, France.
authors.forename: A L
authors.initials: AL
authors.lastname: Perrier
mesh_terms: D000818:Animals; D001787:Blood Group Incompatibility / Q000276:immunology; D018562:Disease Transmission, Infectious; D016332:Fetal Tissue Transplantation / Q000009:adverse effects / Q000379:methods / Q000639:trends*; D006801:Humans; D006816:Huntington Disease / Q000628:therapy*; D058953:Neural Stem Cells / Q000637:transplantation; D039904:Pluripotent Stem Cells / Q000637:transplantation*; D044968:Regenerative Medicine / Q000379:methods / Q000639:trends; D033581:Stem Cell Transplantation / Q000009:adverse effects / Q000639:trends
keywords: Cell therapy; Huntington's disease; Maladie de Huntington; Thérapie cellulaire