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MICROBIOME

Targeting microbial metabolites to treat autism

Nature Medicine volume 28, pages 448–450 (2022)Cite this article

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A first-in-class therapeutic that targets neuroactive microbial metabolites in the gut shows promising target engagement, safety and behavioral improvements in adolescents with autism spectrum disorder.

Autism spectrum disorder (ASD) is a group of heterogenous neurodevelopment conditions, characterized by deficits in social communication and interaction in conjunction with restricted, repetitive patterns of behaviors and interests. Many affected individuals experience gastrointestinal (GI) dysfunction, as well as a range of comorbidities including sleep disorders, epilepsy and anxiety. Currently, there are no approved drugs for treating the core symptoms of ASD. Although the etiology remains poorly understood, it is widely recognized that genetic and environmental factors and their interactions contribute to ASD phenotypes. One such environmental risk factor is the gut microbiome, a key regulator of brain development and behavior1.

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Fig. 1: Treatment with AB-2004 improves gastrointestinal problems and non-core behavioral symptoms of ASD.

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Acknowledgements

R.D.H. is supported by the Swedish Research Council, the Swedish Brain Foundation, the Frimurare Barnhus Foundation and the European Union (ITN SmartAge, grant agreement number 859890). P.G. is supported by Inserm, the Université de Paris, the Horizon 2020 Framework Program of the European Union (grant agreement no. 874721/PREMSTEM), the ANR, the Fondation Grace de Monaco, the Fondation des Gueules Cassées and an additional grant from “Investissement d’Avenir-ANR-11-INBS-0011”–NeurATRIS. J.R.S. is supported by the National Institute for Health Research Southampton Biomedical Research Centre, Biotechnology and Biological Sciences Research Council (BB/W00139X/1) and Medical Research Council (MR/W003597/1).

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Authors and Affiliations

  1. Department of Neuroscience, Karolinska Institutet, Biomedicum, Stockholm, Sweden

    Rochellys Diaz Heijtz, Pierre Gressens & Jonathan R. Swann

  2. University of Rouen Normandy, INSERM U1239, Rouen, France

    Rochellys Diaz Heijtz

  3. Université de Paris, NeuroDiderot, Inserm, Paris, France

    Pierre Gressens

  4. School of Human Development and Health, Faculty of Medicine, University of Southampton, Southampton, UK

    Jonathan R. Swann

Authors
  1. Rochellys Diaz Heijtz
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  2. Pierre Gressens
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  3. Jonathan R. Swann
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Correspondence to Rochellys Diaz Heijtz.

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Diaz Heijtz, R., Gressens, P. & Swann, J.R. Targeting microbial metabolites to treat autism. Nat Med 28, 448–450 (2022). https://doi.org/10.1038/s41591-022-01711-8

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