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Towards treatments targeting the gut to improve behavioural outcomes in autism spectrum disorder
Mst Shirajum Munira1, Julie E Stevens2,3, Wejdan Shahin2
1Department of Medical Sciences, Gut-Brain Axis Laboratory, School of Health and Biomedical Sciences, RMIT University, Bundoora, VIC, 3083, Australia.
Insights
Investigational drugs and therapies are being trialled to address irritability, social communication deficits, and gastrointestinal symptoms in autism spectrum disorder (ASD). These include repurposed medications, cannabis-derived biopharmaceuticals, and gut-targeted interventions.
Area of Science:
- Neurodevelopmental Disorders
- Gastroenterology
- Pharmacology
Background:
- Autism spectrum disorder (ASD) is a prevalent neurodevelopmental disorder characterized by social communication difficulties and repetitive behaviors.
- Irritability and aggressive behaviors in individuals with ASD can significantly impair quality of life and access to services.
- Gastrointestinal (GI) symptoms are frequently associated with behavioral challenges in ASD, yet treatment options are limited.
Purpose of the Study:
- To review emerging drugs and therapies in clinical trials for ASD.
- To explore treatments targeting irritability, social communication, and GI symptoms in autism.
- To summarize the mechanisms of action and trial status of novel autism interventions.
Main Methods:
- Review of ongoing clinical trials for autism spectrum disorder treatments.
- Analysis of repurposed medications, novel biopharmaceuticals, and gut-targeted therapies.
- Examination of emerging findings on microbial and FMT-based interventions.
Main Results:
- Several repurposed drugs (pimavanserin, suramin, brexpiprazole, lumateperone) and novel agents (NTI164, ML-004, AB-2004) are in clinical trials for ASD symptoms.
- Investigational drugs aim to treat irritability, improve social communication, and address GI dysfunction.
- Emerging research explores gut-targeted therapies including probiotics, prebiotics, bacteriophages, and FMT for behavioral improvement.
Conclusions:
- A range of pharmaceutical and gut-targeted interventions show promise for managing challenging behaviors and GI symptoms in autism.
- Clinical trials are crucial for evaluating the efficacy and safety of these novel treatments for autism spectrum disorder.
- Future research may lead to approved therapies addressing the multifaceted needs of individuals with ASD.
Abstract:
Autism spectrum disorder (ASD; autism) is a prevalent and heterogeneous neurodevelopmental disorder characterised by social communication difficulties, repetitive behaviour, and restricted interests. For individuals with autism, in particular those who require substantial care-giver support, irritability, heightened sensitivity and aggressive behaviours in response to sensory, social, or environmental triggers can limit access to health, education and community services and impact quality of life. Although gastrointestinal (GI) symptom severity is associated with irritable behaviours in autism, there are few approved medications to address challenging behaviour or comorbid psychiatric disorders, or gut dysfunction in autism. Here, we review the mode of action of drugs undergoing clinical trials for treating irritable behaviour and improving social communication as well as potentially gastrointestinal symptoms in individuals with autism. Repurposed medications such as pimavanserin (an atypical antipsychotic) and the antiparasitic suramin are being trialled for treating irritable behaviours and impaired social interaction, respectively, in autism. NTI164 is a medicinal cannabis-derived biopharmaceutical undergoing clinical safety and efficacy trials for improving social communication and similarly, ML-004 is an investigational drug being assessed for treating social communication deficits. Two other repurposed medications previously utilised for schizophrenia; brexpiprazole and lumateperone, as well as AB-2004, a microbial metabolite sequestering agent (with proposed actions on gut function), are undergoing clinical trials to assess impacts on irritability associated with autism. We also outline emerging findings from clinical studies on the use of gut-targeted small molecules and bacteriophage therapy, prebiotics, probiotic supplementation and faecal microbiota transplantation (FMT), and their potential impact on behavioural symptoms in autism.
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