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: A-C Bachoud-Lévi, A L Perrier
• Journal: Revue neurologique (Rev Neurol (Paris)) Vol. 170 Issue 12 Pg. 749-62 (Dec 2014) ISSN: 0035-3787 [Print] France
• PMID: 25459124 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
• Copyright: Copyright © 2014. Published by Elsevier Masson SAS.

Topics

• Animals
• Blood Group Incompatibility (immunology)
• Disease Transmission, Infectious
• Fetal Tissue Transplantation (adverse effects, methods, trends)
• Humans
• Huntington Disease (therapy)
• Neural Stem Cells (transplantation)
• Pluripotent Stem Cells (transplantation)
• Regenerative Medicine (methods, trends)
• Stem Cell Transplantation (adverse effects, trends)