The NSW Government has committed $3.5 million towards research into the use of cannabis medicines for children with severe, treatment-resistant refractory epilepsy. A Compassionate Access Scheme and clinical trials are underway.
Compassionate Access Scheme: treatment now underway
In an Australian first, from July 2016, NSW children with severe, treatment-resistant refractory epilepsy have been provided with compassionate access to Epidiolex®, an oral pharmaceutical formulation of cannabis medicine containing the non-psychoactive cannabinoid - Cannabidiol (CBD), developed by the UK-based company GW Pharmaceuticals, a world leader in the development of prescription cannabis medicines. Epidiolex® has shown promise in the treatment of epilepsy in children and young people in overseas Phase III clinical trials.
The Compassionate Access Scheme is for a small number of children who are not able to participate in Paediatric Epilepsy clinical trials due to the unrelenting nature of their epilepsy, which has proven unresponsive to all conventional treatments. As of October 2017, 64 children have now received Epidiolex® and the recent delivery of an additional 26 doses means that up to 66 of NSW’s sickest children at any one time will be able to gain access to this cannabis medicine.
NSW researchers at the Sydney Children’s Hospitals Network (SCHN) are leading the Compassionate Access Scheme (see accompanying Fact Sheet) and clinical trials. These are currently underway across four sites: Sydney Children’s Hospital, Randwick; The Children’s Hospital at Westmead; Royal North Shore Hospital and John Hunter Children’s Hospital. While providing potential relief from the symptoms of severe refractory epilepsy for sick children and their families, the NSW Government’s Compassionate Access Scheme for Epidiolex® is also providing valuable safety information, which adds to the international evidence base for the treatment of childhood epilepsy.
NSW joins two international trials
Australia’s relatively small population, combined with the rarity of some conditions, means that establishment of clinical trials for certain conditions are difficult. However, the GW Pharmaceuticals partnership has allowed NSW children and young people with rare conditions to join international trials to access treatment that they would not otherwise have been able to receive.
The SCHN is taking part in one such GW-sponsored international clinical trial evaluating the use of Epidiolex® in treating seizures in people suffering from the rare disorder Tuberous Sclerosis Complex (TSC). This Phase III global study, allows 200 patients access to Epidiolex®, in which 10 children in NSW are participating, through the SCHN, Clinical Research Centre.
Through the SCHN, NSW children are taking part in a further international trial evaluating the use of Epidiolex® in treating Dravet Syndrome in both children and young adults, also being sponsored by GW Pharmaceuticals.
NSW world-first trial in final planning stage
Another clinical trial, funded by the NSW Government and designed by the SCHN, aims to evaluate the long-term safety and efficacy of a new cannabis medicine - Cannabidivarin (CBDV). Cannabidivarin will be used to treat seizures in 30 children with Rett Syndrome aged between the ages of one and 18 years.
All trials are being conducted in accordance with Good Clinical Practice for clinical trials and are subject to regulatory approval, including review and approval by an appropriate Human Research Ethics Committee and Research Governance Office.
Participants are referred to the trials according to an inclusion and exclusion criteria and participant numbers are calculated based on expert statistical analysis to achieve a scientifically valid outcome.
Suitability for referral to the Compassionate Access Scheme or one of these trials should be discussed with the child’s neurologist.