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MDS 2021 | Phase I trial of fetal striatal cell transplantation in Huntington’s disease

Huntington’s disease is characterized by loss of striatal medium spiny neurons, making cell-replacement therapy a suitable candidate therapeutic approach. Anne Rosser, PhD, FRCP, Cardiff University, Cardiff, UK, discusses the launch of an upcoming study investigating the safety and feasibility of human fetal striatal cell transplantation in Huntington’s disease (ISRCTN52651778). Transplantation of fetal cells has been shown to enhance repair of the striatum in animal models of Huntington’s disease. Up to 5 participants will undergo transplantation in a dose-escalation paradigm. Safety assessments will be conducted 4 weeks after surgery and detailed clinical follow-up will continue for a minimum of 12 months. Prof. Rosser also discusses the derivation of donor cells from pluripotent stem cell sources and the extent to which these cells resemble medium spiny neurons derived from the fetal brain. This interview took place during the 2021 International Congress of Parkinson’s Disease and Movement Disorders.