Paediatric epilepsy
The NSW Government has committed $3.5 million towards research into the use of cannabis medicines for children with severe, treatment-resistant refractory epilepsy, including a Compassionate Access Scheme and clinical trials.
Compassionate Access Scheme
In July 2016, the NSW Government established a scheme to give NSW children with severe, treatment-resistant refractory epilepsy compassionate access to Epidiolex (subsequently registered on the Australian Register of Therapeutic Goods as Epidyolex®), an oral pharmaceutical formulation of cannabis medicine containing the non-psychoactive cannabinoid Cannabidiol (CBD).1 Epidyolex® was developed by the UK-based company GW Pharmaceuticals and has shown promise in the treatment of epilepsy2,3 in children and young people.
The Compassionate Access Scheme was established for a small number of children unable to participate in paediatric epilepsy clinical trials due to the refractory nature of their epilepsy, which was unresponsive to all conventional treatments. Up to 66 of NSW’s sickest children at any one time could gain access to this cannabis medicine under the Compassionate Access Scheme.
NSW researchers at the Sydney Children’s Hospitals Network (SCHN) led the Compassionate Access Scheme and paediatric epilepsy clinical trials. In addition to providing potential relief from the symptoms of severe refractory epilepsy for sick children and their families, the Compassionate Access Scheme for Epidyolex® contributed to the evidence base which supported it being registered on the Australian Register of Therapeutic Goods in September 2020.
Rett Syndrome clinical trial
A Phase I investigator led clinical trial, funded by the NSW Government and designed by the SCHN, was set up to evaluate the long-term safety and efficacy of a new cannabis medicine called Cannabidivarin (CBDV). CBDV was used to treat seizures in five children in NSW with Rett Syndrome.
International clinical trial – Tuberous Sclerosis Complex
The NSW Government's partnership with GW Pharmaceuticals has allowed three NSW children and young people with the rare disorder Tuberous Sclerosis Complex (TSC) to take part in the GW-sponsored international clinical trial evaluating the use of Epidyolex® in treating their seizures.
International clinical trial – Dravet Syndrome
The GW Pharmaceuticals partnership has also allowed two NSW children to take part in an international trial evaluating the use of Epidyolex® in treating Dravet Syndrome. This trial was sponsored by GW Pharmaceuticals.
Participating in a paediatric epilepsy trial
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. Safety data were reviewed by an independent Data Safety Monitoring Committee. Suitability for referral to the Compassionate Access Scheme or one of these trials should be discussed with the child’s neurologist.
The Compassionate Access Scheme has now closed recruitment following Therapeutic Goods Administration approval of Epidyolex®. The clinical trials for Rett Syndrome, Dravet Syndrome and Tuberous Sclerosis Complex have closed for recruitment.
References
1 Cannabidiol for treating drug-resistant epilepsy in children: the New South Wales experience Kerrie-Anne Chen, Michelle Farrar, Michael Cardamone, Deepak Gill, Robert Smith, Christopher T Cowell, Linda Truong, John A Lawson, MJA Research, 2018, 219 -221
2 Thiele, E, Marsh, E, Mazurkiewicz‐Beldzinska, M, et al. Cannabidiol in patients with Lennox‐Gastaut syndrome: Interim analysis of an open‐label extension study. Epilepsia. 2019; 60: 419– 428.
3 Trial of cannabidiol for drug-resistant seizures in the Dravet syndrome; New England Journal of Medicine (05/25/17) Vol. 376, No. 21, P. 2011 Devinsky, Orrin; Cross, J. Helen; Laux, Linda; et al.