What topics does this cannabis study cover?

This study covers research on: Mental Health, CBD, THC, Inflammation, Neuroprotection, Endocannabinoid.

Mental Health CBD THC Inflammation Neuroprotection Endocannabinoid

Low-THC cannabis shows disease-modifying potential for kids

Q: What did this research study find?

**12 weeks** of *low-THC cannabis extract* treatment reduced overall disease severity scores from **4.8 to 3.3** (p = 0.002) in children with chronic neuropsychiatric syndrome

Q: Where was this research published?

Published in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics on January 8, 2026.

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

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics • January 8, 2026 • Highly Relevant

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AI Summary

A groundbreaking open-label trial investigated whether a cannabinoid-rich, low-THC cannabis extract called NTI164 could help children suffering from Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS), a devastating condition where infections trigger sudden-onset obsessive-compulsive disorder or severe eating restrictions. The study enrolled 14 children (average age 12 years) with chronic-relapsing PANS who received 20 mg/kg/day of the cannabis extract for 12 weeks. Researchers used advanced multi-omic analysis—examining gene expression, proteins, and DNA methylation—to understand exactly how the treatment affected patients at a molecular level.

The results were striking: the cannabis extract was well-tolerated and produced significant improvements across multiple symptom measures. The Clinical Global Impression-Severity score dropped from 4.8 to 3.3 (p = 0.002), indicating substantial overall improvement. Children showed remarkable gains in emotional regulation (p < 0.0001), obsessive-compulsive symptoms (p = 0.0001), tics (p < 0.0001), ADHD symptoms (p = 0.028), and quality of life (p = 0.011). Perhaps most importantly, the molecular analysis revealed that patients with PANS had widespread disruptions in epigenetic pathways—the biological switches that control gene expression—along with abnormalities in immune system function and cellular protein production. The cannabis treatment significantly corrected these molecular abnormalities, suggesting it works as a true disease-modifying therapy rather than simply masking symptoms. This research positions epigenetic machinery as a promising therapeutic target and demonstrates that carefully formulated cannabis extracts may address the root biological dysfunctions underlying complex neuropsychiatric conditions in children.

💡 Key Findings

12 weeks of low-THC cannabis extract treatment reduced overall disease severity scores from 4.8 to 3.3 (p = 0.002) in children with chronic neuropsychiatric syndrome

Good

75%

Children experienced highly significant improvements in obsessive-compulsive disorder (p = 0.0001), emotional regulation (p < 0.0001), and tics (p < 0.0001) with the cannabinoid treatment

High

80%

Multi-omic analysis revealed that PANS patients had widespread dysregulation of epigenetic pathways, which the cannabis extract significantly corrected at the molecular level

High

85%

The cannabis extract was well-tolerated with no serious adverse events reported in the 14 pediatric participants over 12 weeks

Good

70%

Treatment modified immune, ribosomal, and signaling pathways, suggesting NTI164 acts as a disease-modifying therapy rather than simply treating symptoms

Good

75%

📄 Original 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.

Paper Type

📄 Research

Publication

Journal: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
Published: January 8, 2026
PMID: 41513541
DOI: 10.1016/j.neurot.2025.e00828

Authors (15)

Brooke A Keating Kids 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 Han Kids 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 Nishida Kids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia.
Nader Aryamanesh Bioinformatics 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 Marshall Bioinformatics Group, Children's Medical Research Institute, University of Sydney, Westmead, NSW, Australia.
+10 more authors