Medicinal Cannabis: Positive results in treatment of children with seizures from Tuberous Sclerosis Complex (TSC)

03 June 2019
Medicinal Cannabis

Medicinal Cannabis: Positive results in treatment of children with seizures from Tuberous Sclerosis Complex (TSC)

A phase 3 clinical trial has shown that the cannabis-based medicine Epidiolex ® (cannabidiol oral solution or CBD) manufactured and supplied by GW pharmaceuticals was successful in the treatment of seizures associated with Tuberous Sclerosis Complex (TSC).

TSC is a rare genetic disorder that is characterised by the growth of benign tumours in the brain, kidneys, lungs, skin and other organs. With epilepsy and seizures being one of the major symptoms of the disorder - expanding knowledge of this newly available medicine and its potential as a treatment is crucial.

Sydney Children’s Hospital, Randwick with Dr John Lawson, paediatric neurologist as primary investigator was one of the lead sites in this international study which investigated the safety and efficacy of the drug in reducing seizures in children with the condition.

In this trial, 224 participants were randomized, with 75 participants given Epidiolex ® of 25mg/kg/day, 73 participants given Epidiolex ® of 50mg/kg/day or 76 patients with placebo was added to their current anti-epileptic drug (AED) treatment.

The trial showed positive results where Epidiolex ® was successful in reducing the frequency of TSC associated seizures over the 16-week treatment period (4-week titration followed by 12-week maintenance at the target dose). Participants who were given Epidiolex ® saw a reduction in TSC associated seizures of nearly 50 percent compared to a near 27 percent reduction in patients taking placebo, which is a significant improvement for patients experiencing seizures regularly each day.

Clinical trials of new medicines in rare diseases are a vital role for Kids Research at SCHN. 

Full media release can be found here.

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