Cannabis-based oil eases symptoms in Rett syndrome girls
Neurotech's NTI164 may improve communication ability, quality of life
NTI164, a cannabis-based oil being developed by Neurotech International, may help girls with Rett syndrome communicate more easily, enjoy better moods, and have a better quality of life with few side effects, a small study in Australia found.
The study, “Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label Study,” was published in the Journal of Paediatrics and Child Health. It was funded by Neurotech and its partner Fenix Innovation Group.
Rett syndrome is caused in almost all cases by mutations in the MECP2 gene, which provides instructions for producing a protein needed for the brain to develop normally. A shortage of the MeCP2 protein disrupts how nerve cells function, leading to seizures and other Rett symptoms.
Rett babies typically develop normally in the first few months of life, but their growth typically begins to lag between 6 and 18 months. Initial symptoms include problems sitting and walking, lack of interest in toys, abnormal hand movements, and speech difficulties.
NTI164 contains a combination of cannabinoids — including a low amount of tetrahydrocannabinol (THC) and other active compounds from cannabis — that protects the brain and reduces inflammation, which may prevent seizures and ease other symptoms.
Symptoms ease, behavior improves for girls in trial
The open-label Phase 1/2 clinical study (NCT06621043) tested oral NTI164 — twice daily at a maximum dose of 20 mg/kg per day — in 11 girls with Rett syndrome with a mean age of 8.8, over 12 weeks (three months). Patients who stopped treatment reduced their dose by 5 mg/kg each week over four weeks. Those continuing could take it for up to about one year, then follow the same gradual reduction.
Doctors and caregivers tracked changes in symptoms using clinical tools including the Clinical Global Impression (CGI) scales and questionnaires designed specifically for symptoms of Rett.
By the end of the first 12 weeks, results showed significantly better communication, increased alertness, improved eye contact and social interaction, and reduced anxiety. These changes were rated using the CGI-Improvement and CGI-Severity scales, which help assess symptom improvement and severity.
Caregivers also reported fewer behavioral problems using the Rett Syndrome Behavior Questionnaire. Responses showed significant reductions in mood issues, abnormal breathing, repetitive facial movements, and anxiety. They also reported improvements in the girls’ ability to communicate choices and in their verbal communication — often difficult for those with Rett.
Quality of life also improved. A tool called ICND+QoL, which measures how neurological diseases affect daily life, showed gains in both thinking ability and overall well-being. Caregivers saw less burden, as shown by a separate scale that tracks the emotional physical and social toll.
Side effects included mild decreased appetite, regurgitation, and vomiting, each occurring in one patient. One patient also experienced a serious allergic reaction deemed related to NTI164. That girl was withdrawn from the study.
While the number of patients was small, the findings were “encouraging” and support further research into how medicinal cannabis could help children with Rett syndrome, the researchers wrote.
“NTI164 demonstrated safety and improved some clinical and functional outcomes in RTT [Rett],” they wrote. “These improvements justify ongoing research into NTI164, which may be a potential adjunct therapy in RTT.”
A larger Phase 2/3 study is being planned in Australia to test NTI164 in more patients with Rett syndrome, according to the researchers. That study will include a wider age range and use a stronger study design testing NTI164 against a placebo.
About the Author
