The Marcus Center for Cellular Cures, Duke University, Durham, NC, USA.
Dev Med Child Neurol. 2021 May;63(5):503-510. doi: 10.1111/dmcn.14789. Epub 2021 Jan 4.
Across disciplines, there is great anticipation that evolving cell therapies may finally provide a therapeutic option for conditions in dire need. These conditions are typically complex and their pathophysiology incompletely understood, hindering the development of robust preclinical models and the precise assessment of therapeutic effects in human studies. This article provides an overview of the status of cell therapy investigations in two common neurodevelopmental disorders, cerebral palsy and autism spectrum disorder. Challenges facing this line of study, including inherent heterogeneity, knowledge gaps, and unrealistic expectations, are discussed. Much progress has been made in the past decade, but to definitively determine if cell therapies have a role in the treatment of neurodevelopmental disorders, both fields will need to evolve together. WHAT THIS PAPER ADDS: The safety profile of reported cell therapies in children with neurodevelopmental disorders is encouraging. Efficacy trials in cerebral palsy and autism spectrum disorder are ongoing in the United States and Asia. Unresolved issues pertain to the properties of the cells being studied and the characteristics of the neurodevelopmental conditions themselves.
跨学科领域普遍期待细胞疗法的不断发展最终可为一些亟需治疗的疾病提供治疗选择。这些疾病通常较为复杂，其病理生理学机制尚未完全阐明，这阻碍了稳健的临床前模型的开发以及在人体研究中对治疗效果的精确评估。本文概述了细胞疗法在两种常见神经发育障碍（脑瘫和自闭症谱系障碍）中的研究现状。该研究领域面临的挑战包括固有异质性、知识空白和不切实际的期望，对此进行了讨论。过去十年已取得诸多进展，但要明确确定细胞疗法是否对神经发育障碍的治疗有作用，这两个领域都需要共同发展。本文的贡献在于：报道的神经发育障碍儿童细胞疗法的安全性概况令人鼓舞。脑瘫和自闭症谱系障碍的疗效试验正在美国和亚洲进行。未解决的问题涉及到所研究细胞的特性以及神经发育障碍本身的特征。
