NTI has reported more positive results for its broad spectrum cannabinoid drug. Pic: Getty Images.
Neurotech’s cannabinoid drug reverses immune dysregulation in PANDAS/PANS kids
Health & Biotech
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- Neurotech reports positive genomic analysis of NTI164 as part of Phase 1/2 PANDAS/PANS trial
- NTI164 shown to positively modify immune cell function and address gene translation dysregulation
- Disease-modifying potential of NTI164 may improve outcomes in children with PANDAS/PANS
Special Report: Neurotech International has announced positive results of a genomic analysis conducted as part of its Phase 1/2 open-label clinical trial involving 15 paediatric patients treated with its broad-spectrum cannabinoid drug NTI164.
The Phase 1/2 trial is focused on children diagnosed with Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS).
Neurotech International (ASX:NTI) said new proteomic insights into the biological effects of NTI164 in PANDAS/PANS patients correlate to the significant clinical benefits observed in the Phase 1/2 clinical trial at 12 weeks.
The clinical-stage biopharmaceutical focused predominantly on paediatric neurological disorders said NTI164 had been shown to positively modify immune cell function and address gene translation dysregulation, improving the overall health and functional outcomes of children.
Certain important dysregulated pathways associated with immune function and gene translation, including MECP2, were significantly reversed with NTI164.
MECP2 is the defective gene in Rett Syndrome, which NTI is also targeting with NTI164. NTI recently reported positive results from the extension phase of Phase 1/2 trial of NTI164 to treat Rett Syndrome.
PANDAS/PANS is a rare neurological disorder predominantly in children characterised by an infection triggered autoimmune response and associated neuroinflammation, which results in a sudden, dramatic change in personality, displayed as obsessive-compulsive disorder, anxiety, tics or other abnormal movements and personality changes.
NTI said the disease-modifying potential of NTI164 may improve outcomes in children with PANDAS/PANS by normalising their immune function and gene translation profiles.
Disease modifying treatment
NTI said proteome is the complete set of proteins expressed by an organism (in this case, each child). Proteomics analysis allows for the study of interactions, functions, and changes in the expression of these proteins over time.
In NTI’s proteomics analysis, the quantity of all proteins in PANDAS/PANS children in its trial was measured and compared to controls, which were healthy children without neurodevelopmental disorders.
The analysis revealed that NTI164 can modulate the expression of genes related to immune cell function in PANDAS/PANS patients, suggesting that the treatment can adjust the immune system’s response.
NTI said PANDAS/PANS patients have dysregulation in two key areas – gene translation (the process of converting genetic information into proteins) and immune function.
The genomic analysis showed NTI164 treatment was found to improve both areas, helping to restore normal function.
NTI further discovered that NTI164 impacts the phosphoproteome, which is the set of proteins in a cell that have undergone phosphorylation.
“Phosphorylation modification plays a crucial role in regulating protein function, activity, interactions, localisation, and stability, and is involved in numerous cellular processes such as signal transduction, cell cycle control, and metabolism,” NTI said.
“The phosphoproteome of patients after 12 weeks of NTI164 showed a significant normalisation towards matched healthy normal controls.”
NTI said researchers concluded that PANDAS/PANS patients have inflammatory cellular environments due to gene dysregulation which is normalised with NTI164 treatment.
“NTI164 appears to have significant anti-inflammatory effects as well as potential as an epigenetic modulator.”
Showing promise to modify ‘debilitating condition’
Professor Russell Dale – professor of paediatric neurology, University of Sydney and Children’s Hospital at Westmead and co-principal investigator of the NTIPANS1 trial – said the results back up his research group’s hypothesis.
“My research group hypothesises that PANDAS/PANS is the result of gene-environment (epigenetc) neuroimmune dysregulation leading to persistent or progressive neuroinflammation,” he noted.
“Currently most patients are symptomatically managed, whereas NTI164’s anti-neuroinflammatory properties have now been shown to induce important epigenetic and proteomic changes in immune cells collected from patients at baseline (day zero) and after 12 weeks of treatment.
“This data is exciting as it demonstrates that the biological aspects of this debilitating condition can be modified by NTI164, including immune and epigenetic dysregulation, improving overall health and functional outcomes of children with PANDAS/PANS.”
Dale said his team eagerly awaited further genomic analysis from these same patients.
This article was developed in collaboration with Neurotech a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
