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Review
. 2021 Jul:48:3-31.
doi: 10.1016/j.euroneuro.2021.05.010. Epub 2021 Jun 19.

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

James T McCracken  1 , Evdokia Anagnostou  2 , Celso Arango  3 , Geraldine Dawson  4 , Tiffany Farchione  5 , Valentina Mantua  5 , James McPartland  6 , Declan Murphy  7 , Gahan Pandina  8 , Jeremy Veenstra-VanderWeele  9 ; ISCTM/ECNP ASD Working Group
Affiliations
Review

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

James T McCracken et al. Eur Neuropsychopharmacol. 2021 Jul.

Erratum in

Abstract

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.

Keywords: Autism; Biomarkers; Children; Clinical trials; Drug development; Endpoints; Treatment.

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Conflict of interest statement

Disclosures Dr. Anagnostou reports research funding from CIHR, Ontario Brain Institute, Brain Canada, Genome Canada, Ontario Research Fund, Azrieli Foundation, and Autism Speaks. Dr. Arango has been a consultant to or has received honoraria or grants from Acadia, Angelini, Gedeon Richter, Janssen Cilag, Lundbeck, Minerva, Otsuka, Roche, Sage, Servier, Shire, Schering Plough, Sumitomo Dainippon Pharma, Sunovion and Takeda. Dr. Dawson is on the Scientific Advisory Boards of Janssen Research and Development, Akili, Inc, LabCorp, Inc, Roche Pharmaceutical Company, and Tris Pharma, and is a consultant to Apple, Gerson Lehrman Group, Guidepoint, Inc, Axial Ventures, Teva Pharmaceutical, and is CEO of DASIO, LLC. Dr. Dawson has received book royalties from Guilford Press, Oxford University Press, Springer Nature Press. In addition, Dr. Dawson has the following patent applications: 1802952, 1802942, 15141391, and 16493754. Dr. Dawson has developed technology that has been licensed and Dawson and Duke University have benefited financially. Dr. McCracken reports consultant income from Roche, Octapharma, TRIS Pharmaceuticals, GW Biosciences, and research contracts with Octapharma and GW Biosciences. Dr. McPartland consults with Customer Value Partners and BlackThorn Therapeutics, has received research funding from Janssen Research and Development, and receives royalties from Guilford Press, Lambert, and Springer. Dr. Murphy has received honoraria and research funding from Roche. Dr. Pandina is a full-time employee of Janssen Research & Development, LLC, and a Johnson & Johnson stockholder. Dr. Veenstra-VanderWeele reports income from consulting or advisory boards from Roche, Novartis, SynapDx, and research funding from Roche, Novartis, SynapDx, and Wiley. The other authors report no disclosures.

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