Neurotech’s cannabinoid drug shows promise for treatment of PANDAS and PANS

Paediatric neurological disorders specialist Neurotech International (ASX: NTI) has received promising results from its trial of potential treatments for the Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS) conditions.

PANDAS and PANS are disorders that a child can develop following an infection, usually strep, and it has been estimated that 1 in every 200 children may have the disorder.

The conditions may lead to a sudden onset of severe obsessive-compulsive disorder symptoms and in some cases cause other sudden changes in a child’s behaviour.

First a broad-spectrum cannabinoid therapy test

Neurotech has been undertaking what is believed to be the first ever trial to treat the disorders using a broad-spectrum cannabinoid therapy.

To date it has obtained strong results from the 15 patients who participated in an open-label Phase I/II clinical trial of NTI164 involving children diagnosed with PANDAS and PANS.

The NTIPANS1 clinical trial examined safety and gold standard measures of clinical symptoms associated with PANDAS/PANS relating to the severity of their condition, including critical measures relating to anxiety, depression, obsessive compulsive disorders and physical tic movements at 12 weeks compared to baseline measures.

Clinical improvements noted

Professor Russell Dale, professor of paediatric neurology, University of Sydney and Children’s Hospital at Westmead and co-principal investigator of the trial, said NTI164 showed clinically significant and meaningful improvements in clinical function, with excellent safety and tolerability over the 12 weeks of daily oral treatment.

“I am very pleased with the clinical results reported to date and wish to thank all patients and their families for participating in this novel clinical trial. I have observed quite profound improvements in a number of my patients with NTI164, making it the first trial of its kind with a broad-spectrum cannabinoid therapy showing initial clinical utility like this with excellent safety,” he said.

“In addition, we await further evidence of genomic molecular changes from baseline measures and after 12 weeks of treatment to correlate this meaningful clinical response we have seen with biological evidence of effect. This would be a major step-forward for PANDAS/PANS patients and assist in identifying relevant biomarkers of the disease.”

Statistically significant efficacy

Key information gathered from the trial found that NTI164 provided statistically significant efficacy in improving the symptoms associated with PANDAS/PANS after 12 weeks of daily therapy, with the trial meeting the key primary endpoints of anxiety and depression (RCADS-P) with a 30% improvement overall and severity of illness displaying an 18% improvement.

Other efficacy measures also showed improvements over 12 weeks of treatment.

Importantly the trials found NTI164 to be safe and well tolerated.

The trial also concluded there were no serious or severe side-effects and that the treatment did not significantly interfere with patients’ functioning. No clinically significant abnormal laboratory values were reported.

James Fletcher, president of the non-profit organisation PANS Australia and New Zealand Advocacy and Support, said the results were very encouraging.

“As our researchers continue to expand their understanding of PANS, they also help us to raise awareness of PANS within the general medical community. This in turn leads to earlier intervention and better long-term outcomes for children and adults with PANS. A targeted treatment for PANS in the form of NTI164 is a very exciting development indeed. Many thanks to the researchers for their work on this therapy and to the children and families who participated in the study.”

Neurotech says this is the first ever clinical trial to show highly significant clinical improvements in PANDAS/PANS patients with a broad-spectrum cannabinoid drug therapy (NTI164).

The company’s executive director, Dr Thomas Duthy, said statistically significant and clinically meaningful improvements were achieved over a range of gold-standard, clinically validated assessments during the 12 week trial.

“We commenced this clinical trial based on a small number of scientific publications that highlighted recurring, neuroinflammatory processes in these difficult to treat patients.”

“With our established evidence in autism and supportive pre-clinical data we took the decision to run this world-first trial of NTI164 with Professor Dale and Professor Fahey, which has shown very strong benefits for these children over 12 weeks of daily treatment.”

“Given the lack of safe and effective treatments for PANDAS/PANS with associated distressing symptoms and significant caregiver burden we remain very hopeful of an accelerated development plan for NTI164 to bring this therapy to market.”