Cell transplantation for Huntington’s disease: practical and clinical considerations

Abstract

Huntington’s disease is a dominantly inherited neurodegenerative disorder, usually starting in mid-life and leading to progressive disability and early death. There are currently no disease-modifying treatments available. Cell transplantation is being considered as a potential therapy, following proof of principle that cell transplantation can improve outcomes in another basal ganglia disorder, namely Parkinson’s disease. The principle aim is to replace the striatal medium spiny neurons lost in Huntington’s disease with new cells that are able to take over their function and reconnect the circuitry. This article reviews the experimental background and evidence from clinical studies that suggest that cell transplantation may improve function in Huntington’s disease, reviews the current status of the field and considers the current challenges to taking this experimental strategy forward to becoming a reliable therapeutic option.