Bachoud-Lévi A-C, Perrier A L
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.
Inserm U861, I-STEM, AFM, 91030 Évry cedex, France; UEVE U861, I-STEM, AFM, 91030 Évry cedex, France.
Rev Neurol (Paris). 2014 Dec;170(12):749-62. doi: 10.1016/j.neurol.2014.10.007. Epub 2014 Nov 4.
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.
亨廷顿舞蹈症目前无法治愈，但细胞疗法被视为一种有前景的替代治疗方法。我们分析了将来自神经节隆起的人胎儿神经母细胞纹状体内移植到亨廷顿舞蹈症患者体内的安全性和有效性。报告了一些罕见的手术事故，但这种治疗方法的主要困难在于受体对移植物产生同种免疫，以及细胞疗法所需的胎儿源产品缺乏可用性、标准化和质量控制。一些患者在六年多的时间里持续出现认知改善，在四年多的时间里出现运动改善。即使在单个移植中心内，移植结果也存在差异。这种差异的原因尚不清楚，这凸显了在这一特定领域进一步研究的必要性。鉴于未来将进行更多试验，我们在此回顾用于亨廷顿舞蹈症的人多能干细胞衍生细胞治疗产品的发展，以及它们相对于胎儿细胞的优缺点。
