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Study breakdown

Cannabis Extract Shows Promise for Childhood Neuropsychiatric Syndrome Through Epigenetic Mechanisms

Clinical TrialPreliminary evidence
The takeaway

A full-spectrum low-THC cannabis extract (NTI164) significantly improved OCD, tics, ADHD, and emotional regulation in 14 children with PANS, with multi-omics revealing that it modifies the epigenetic and immune dysregulation underlying the condition.

Pediatric neurologists, PANS/PANDAS specialists, parents of children with PANS, immunologists, and epigenetics researchers.

What the researchers found

12 weeks of NTI164 (20 mg/kg/day) decreased CGI-Severity from 4.8 to 3.3 (p=0.002) with significant improvements in OCD (p=0.0001), tics (p<0.0001), ADHD (p=0.028), emotional regulation (p<0.0001), and quality of life (p=0.011). Multi-omics revealed NTI164 modified dysregulated epigenetic, ribosomal, and immune pathways in patient leukocytes.

Why it matters

PANS is a devastating condition with limited treatment options, and this study provides both clinical evidence of benefit and mechanistic evidence that a cannabis extract can modify the underlying epigenetic and immune dysfunction.

The numbers in context

N=14; mean age 12.1; 20 mg/kg/day NTI164; CGI-S: 4.8→3.3 (p=0.002); CYBOCS-II OCD p=0.0001; YGTSS tics p<0.0001; Conner's ADHD p=0.028; RCADS-P emotional p<0.0001; EQ-5D-Y QoL p=0.011; well-tolerated

How the study worked

Open-label trial of 14 children with chronic-relapsing PANS (mean age 12.1, 71% male) receiving NTI164 for 12 weeks, with clinical assessments using gold-standard tools and pre/post blood samples analyzed by bulk and single-cell transcriptomics, proteomics, phosphoproteomics, and DNA methylation.

What this study cannot tell us

Open-label without placebo control; very small sample (n=14); PANS diagnosis can be heterogeneous; cannot separate placebo effects; NTI164 is a complex mixture making mechanism attribution difficult; 12-week duration only.

How to read the evidence

Open-label pilot with multi-omics validation provides compelling preliminary evidence, but lack of placebo control and small sample demand cautious interpretation.

When this study was published

Published 2026; represents cutting-edge integration of cannabinoid therapeutics and multi-omics.

The bigger picture

This is among the first studies to combine clinical outcomes with deep multi-omics profiling to show how a cannabis extract works at the molecular level, presenting epigenetic machinery as a new therapeutic target.

Questions still open

  • Would a placebo-controlled trial confirm these results? Which components of NTI164 drive the epigenetic effects? Could this approach work for other autoimmune neuropsychiatric conditions? What are the long-term effects?

Common questions

Can cannabis help children with PANS?
This small trial of a low-THC cannabis extract showed significant improvements in OCD, tics, ADHD, and emotional regulation in 14 children with PANS — but without a placebo group, these promising results need confirmation in larger controlled studies.
How does cannabis affect PANS at the molecular level?
Multi-omics analysis revealed that PANS patients have dysregulated epigenetic and immune pathways, and the cannabis extract (NTI164) significantly modified these pathways — suggesting it works as a disease modifier rather than just treating symptoms.

Read the original research

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, e00828

Citation

Keating, Brooke A; Han, Velda X; Nishida, Hiroya; Aryamanesh, Nader; Marshall, Lee L; Gloss, Brian S; Lau, Xianzhong; Dissanayake, Ruwani; Dervish, Suat; Graham, Mark E; Mohammad, Shekeeb S; Kanhangad, Manoj; Fahey, Michael C; Patel, Shrujna; Dale, Russell C. (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, e00828. https://doi.org/10.1016/j.neurot.2025.e00828

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