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Drug development for Autism Spectrum Disorder (ASD): Progress, challenges, and future directions.

Publication ,  Journal Article
McCracken, JT; Anagnostou, E; Arango, C; Dawson, G; Farchione, T; Mantua, V; McPartland, J; Murphy, D; Pandina, G; Veenstra-VanderWeele, J ...
Published in: Eur Neuropsychopharmacol
July 2021

In 2017, facing lack of progress and failures encountered in targeted drug development for Autism Spectrum Disorder (ASD) and related neurodevelopmental disorders, the ISCTM with the ECNP created the ASD Working Group charged to identify barriers to progress and recommending research strategies for the field to gain traction. Working Group international academic, regulatory and industry representatives held multiple in-person meetings, teleconferences, and subgroup communications to gather a wide range of perspectives on lessons learned from extant studies, current challenges, and paths for fundamental advances in ASD therapeutics. This overview delineates the barriers identified, and outlines major goals for next generation biomedical intervention development in ASD. Current challenges for ASD research are many: heterogeneity, lack of validated biomarkers, need for improved endpoints, prioritizing molecular targets, comorbidities, and more. The Working Group emphasized cautious but unwavering optimism for therapeutic progress for ASD core features given advances in the basic neuroscience of ASD and related disorders. Leveraging genetic data, intermediate phenotypes, digital phenotyping, big database discovery, refined endpoints, and earlier intervention, the prospects for breakthrough treatments are substantial. Recommendations include new priorities for expanded research funding to overcome challenges in translational clinical ASD therapeutic research.

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Published In

Eur Neuropsychopharmacol

DOI

EISSN

1873-7862

Publication Date

July 2021

Volume

48

Start / End Page

3 / 31

Location

Netherlands

Related Subject Headings

  • Psychiatry
  • Phenotype
  • Humans
  • Drug Development
  • Communication
  • Biomarkers
  • Autism Spectrum Disorder
  • 5202 Biological psychology
  • 3209 Neurosciences
  • 17 Psychology and Cognitive Sciences
 

Citation

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ICMJE
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McCracken, J. T., Anagnostou, E., Arango, C., Dawson, G., Farchione, T., Mantua, V., … ISCTM/ECNP ASD Working Group, . (2021). Drug development for Autism Spectrum Disorder (ASD): Progress, challenges, and future directions. Eur Neuropsychopharmacol, 48, 3–31. https://doi.org/10.1016/j.euroneuro.2021.05.010
McCracken, James T., Evdokia Anagnostou, Celso Arango, Geraldine Dawson, Tiffany Farchione, Valentina Mantua, James McPartland, et al. “Drug development for Autism Spectrum Disorder (ASD): Progress, challenges, and future directions.Eur Neuropsychopharmacol 48 (July 2021): 3–31. https://doi.org/10.1016/j.euroneuro.2021.05.010.
McCracken JT, Anagnostou E, Arango C, Dawson G, Farchione T, Mantua V, et al. Drug development for Autism Spectrum Disorder (ASD): Progress, challenges, and future directions. Eur Neuropsychopharmacol. 2021 Jul;48:3–31.
McCracken, James T., et al. “Drug development for Autism Spectrum Disorder (ASD): Progress, challenges, and future directions.Eur Neuropsychopharmacol, vol. 48, July 2021, pp. 3–31. Pubmed, doi:10.1016/j.euroneuro.2021.05.010.
McCracken JT, Anagnostou E, Arango C, Dawson G, Farchione T, Mantua V, McPartland J, Murphy D, Pandina G, Veenstra-VanderWeele J, ISCTM/ECNP ASD Working Group. Drug development for Autism Spectrum Disorder (ASD): Progress, challenges, and future directions. Eur Neuropsychopharmacol. 2021 Jul;48:3–31.
Journal cover image

Published In

Eur Neuropsychopharmacol

DOI

EISSN

1873-7862

Publication Date

July 2021

Volume

48

Start / End Page

3 / 31

Location

Netherlands

Related Subject Headings

  • Psychiatry
  • Phenotype
  • Humans
  • Drug Development
  • Communication
  • Biomarkers
  • Autism Spectrum Disorder
  • 5202 Biological psychology
  • 3209 Neurosciences
  • 17 Psychology and Cognitive Sciences