autism spectrum disorder (ASD) is a complex neurodevelopmental condition with uncertain origins. Understanding of the mechanisms underlying ASD re-mains 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.
Pedini, G., Chen, C., Achsel, T., Bagni, C. (2023). Cancer drug repurposing in autism spectrum disorder. TRENDS IN PHARMACOLOGICAL SCIENCES, 44(12), 963-977 [10.1016/j.tips.2023.09.008].
Cancer drug repurposing in autism spectrum disorder
Pedini, Giorgia;Bagni, Claudia
2023-12-01
Abstract
autism spectrum disorder (ASD) is a complex neurodevelopmental condition with uncertain origins. Understanding of the mechanisms underlying ASD re-mains 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.File | Dimensione | Formato | |
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