Evidence map›Paper›PMID 41513541›Full record

Trial reportNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2026

Medicinal cannabis plant extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in paediatric acute-onset neuropsychiatric syndrome.

Brooke A Keating, Velda X Han, Hiroya Nishida, Nader Aryamanesh, Lee L Marshall, Brian S Gloss, Xianzhong Lau, Ruwani Dissanayake, Suat Dervish, Mark E Graham and 5 more

Abstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Brooke A KeatingKids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; Fenix Innovation Group Pty Ltd, Mount Waverley, VIC 3149, Australia.
Velda X HanKids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; Khoo Teck Puat-National University Children's Medical Institute, National University Health System, Singapore, Singapore; Department of Paediatrics, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.
Hiroya NishidaKids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia.
Nader AryamaneshBioinformatics Group, Children's Medical Research Institute, University of Sydney, Westmead, NSW, Australia; Faculty of Medicine and Health, Sydney Medical School, University of Sydney, Camperdown, NSW, Australia.
Lee L MarshallBioinformatics Group, Children's Medical Research Institute, University of Sydney, Westmead, NSW, Australia.
Brian S GlossWestmead Research Hub, Westmead Institute for Medical Research, Westmead, NSW, Australia.
Xianzhong LauAustralian Genome Research Facility Ltd, Melbourne, VIC, Australia.
Ruwani DissanayakeAustralian Genome Research Facility Ltd, Westmead, NSW, Australia.
Suat DervishWestmead Institute for Medical Research, Sydney, Australia.
Mark E GrahamBiomedical Proteomics, Children's Medical Research Institute, The University of Sydney, NSW, Australia.
Shekeeb S MohammadKids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; The Children's Hospital at Westmead Clinical School, Faculty of Medicine and Health, University of Sydney, Sydney, NSW, Australia.
Manoj KanhangadDepartment of Neurology, Monash Children's Hospital, Monash Health, Clayton, VIC, Australia.
Michael C FaheyDepartment of Neurology, Monash Children's Hospital, Monash Health, Clayton, VIC, Australia.
Shrujna PatelKids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; The Children's Hospital at Westmead Clinical School, Faculty of Medicine and Health, University of Sydney, Sydney, NSW, Australia.
Russell C DaleKids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; The Children's Hospital at Westmead Clinical School, Faculty of Medicine and Health, University of Sydney, Sydney, NSW, Australia; The University of Sydney, School of Medical Sciences and Discipline of Child and Adolescent Health, Faculty of Medicine and Health, Sydney, NSW, Australia. Electronic address: russell.dale@sydney.edu.au.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Paediatric acute-onset neuropsychiatric syndrome (PANS) is a syndrome of infection-provoked abrupt-onset obsessive-compulsive disorder (OCD) or eating restriction. Based on the hypothesis that PANS is an epigenetic disorder of immune and brain function, a full-spectrum medicinal cannabinoid-rich low-THC cannabis (NTI164) was selected for its known epigenetic and immunomodulatory properties. This open-label trial of 14 children with chronic-relapsing PANS (mean age 12·1 years; range 4-17; 71 % male) investigated the safety and efficacy of 20 mg/kg/day NTI164 over 12 weeks. Clinical outcomes were assessed using gold standard tools. To define the biological effects of NTI164, blood samples were collected pre- and post-treatment for bulk and single-cell transcriptomics, proteomics, phosphoproteomics, and DNA methylation. NTI164 was well-tolerated, and 12 weeks of treatment decreased the mean Clinical Global Impression-Severity (CGI-S) score from 4·8 to 3·3 (p = 0·002). Significant improvements were observed in emotional regulation (RCADS-P, p < 0·0001), obsessive-compulsive disorder (CYBOCS-II, p = 0·0001), tics (YGTSS, p < 0·0001), attention-deficit hyperactivity disorder (Conner's, p = 0·028), and overall quality of life (EQ-5D-Y, p = 0·011). At baseline, the multi-omic approach revealed that leucocytes from patients with PANS had dysregulated epigenetic (chromatin structure, DNA methylation, histone modifications, transcription factors), ribosomal, mRNA processing, immune, and signalling pathways. These pathways were significantly modulated by NTI164 treatment. NTI164 shows promise as a disease-modifying therapeutic for PANS. Multi-omics reveal broad epigenetic and immune dysregulation in patients, which was modified by NTI164, presenting epigenetic machinery as a therapeutic target in PANS.

Indexed as

Autoimmune DiseasesCannabisEpigenesis, GeneticObsessive-Compulsive DisorderPlant ExtractsAdolescentChildChild, PreschoolFemaleHumansMalePlant ExtractsCannabisEpigeneticsImmuneMulti-omicsPANSRibosome

Identifiers

PMID41513541
PMCPMC12976522

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.