自闭症谱系障碍 (ASD) 是一种起源不确定的复杂神经发育疾病。对 ASD 潜在机制的了解仍然有限，并且缺乏治疗方法。遗传多样性使药物开发变得复杂。鉴于 ASD 症状的复杂性和严重性以及诊断数量的不断增加，探索新的治疗策略至关重要。在这里，我们重点关注自闭症谱系障碍 (ASD) 与癌症之间共有的分子通路，并重点介绍用于自闭症谱系障碍 (ASD) 治疗的癌症药物的重新利用的最新进展，例如 mTOR 抑制剂、组蛋白脱乙酰酶抑制剂和抗炎药。我们讨论如何考虑药物剂量和患者年龄来改进试验设计。最后，讨论探讨了药物筛选流程的副作用、商业因素和效率等关键方面；所有这些都是寻求重新利用癌症药物以解决 ASD 核心特征的重要考虑因素。版权所有 © 2023 Elsevier Ltd。保留所有权利。

Autism spectrum disorder (ASD) is a complex neurodevelopmental condition with uncertain origins. Understanding of the mechanisms underlying ASD remains limited, and treatments are lacking. Genetic diversity complicates drug development. Given the complexity and severity of ASD symptoms and the rising number of diagnoses, exploring novel therapeutic strategies is essential. Here, we focus on shared molecular pathways between ASD and cancer and highlight recent progress on the repurposing of cancer drugs for ASD treatment, such as mTOR inhibitors, histone deacetylase inhibitors, and anti-inflammatory agents. We discuss how to improve trial design considering drug dose and patient age. Lastly, the discussion explores the critical aspects of side effects, commercial factors, and the efficiency of drug-screening pipelines; all of which are essential considerations in the pursuit of repurposing cancer drugs for addressing core features of ASD.Copyright © 2023 Elsevier Ltd. All rights reserved.