Drug discovery and development and precision medicine for Autism Spectrum Disorder – current state

Abstract

Introduction: Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder/condition. Medical interven-tions for this condition are mainly pharmacological, and generally not tailored to precisely address the specific underlying issues in each ASD individual. Despite the extensive efforts to develop new or repurpose existing drugs over the decades, the range of medications that address this condition remains very limited.Findings: Progress in drug development has been hindered by research design limitations and the complex, het-erogeneous nature of the ASD itself. Therefore, this article first discusses preclinical and clinical studies aimed at finding effective treatments, highlighting their shortcomings and potential solutions. It then delves into the com-plexity of ASD and the implications for drug development, such as its phenotypic and genetic heterogeneity and multifactorial etiology, and unclear diagnostic boundaries with other developmental disorders.Exploiting the advantages of new technologies, current autism treatment research is steering towards prioritizing genetic and molecular data over phenotypic data, emphasizing the need for biologically meaningful and quantifi-able biomarkers to identify biologically defined and clinically actionable subgroups within ASD, amenable to specific treatments.The critical role of precision medicine is underscored as a comprehensive, fundamental approach to biology-based drug development and personalized treatments. Achieving this goal requires an integrated analysis of multilayered data, utilizing multi-omics, systems biology, and machine learning approaches.Conclusion: Lastly, the article provides a brief overview of current initiatives and private sector efforts focusing on precision medicine treatments for neurodevelopmental disorders, highlighting their progress in developing drugs through this innovative approach.