Cannabis-based epilepsy drug for children at clinical trial stage


A new drug for children suffering severe epilepsy is showing signs of success during the clinical trials.

Epidiolex is currently being tested on children suffering with Dravet Syndrome and other types of epilepsy which is non-responsive to existing drugs.

GW Pharmaceuticals, the manufacturer of the drug, said most of the 60 children participating in the trials thus far have experienced a decline in both convulsive and ‘drop’ seizures.

The most common side effects being experienced are fatigue and sleepiness in around a tenth and a fifth of the patients, respectively.

The director of the Paediatric Epilepsy Programme at Massachusetts General Hospital, Dr Elizabeth Thiele, felt encouraged by the preliminary results. She said that Epidiolex could become an important advancement in treatment for children who are treatment-resistant and will play a huge role in future therapy.

The drug does not have any intoxicating effects. It is a liquid which is made from a purified cannabidiol which is extracted from the marijuana plants that are grown under licence at a secret location within Britain.

The drug’s status was fast-tracked by the US Food and Drug Administration to allow for earlier trials to commence. Placebo-controlled clinical trials for Lennox-Gastaut Syndrome and Dravet Syndrome are due to commence shortly.

Both forms of epilepsy commence during childhood and are extremely difficult to treat.

Of the 151 patients who participated in safety testing for the drug, 26 suffered severe adverse effects and two did, but GW Pharmaceuticals have stated that independent investigations discovered that their deaths were unrelated to the trial.

The participants in the study, with an average of 11, were all on other treatments along with the Epidiolex. Two were removed from the study due to ‘adverse effects’ and four others were removed as they were experiencing ‘a lack of clinical effect’.

The patients were aged between one and 18 and had illnesses which were resistant to many or all of the available anti-epilepsy treatments, including drugs and ketogenic diets.

Maria Roberta Cilio is testing the drug at the Benioff Children’s Hospital in San Francisco and said that the studies which were done on animals showed that cannabidiol acts as an anticonvulsant.

She said it is important to get seizure control at any age, but in children, uncontrolled seizures could affect their brain and neurocognitive development, which will affect their quality of life and lead to progressive cognitive impairment.

She added that the trial is to try and find a new treatment for children suffering with the most severe forms of epilepsy, for which there is no effective treatment.

However, she said the trials are in its early stages.

Epidiolex has only been used for testing on American children thus far, but trials are expected to commence in Europe soon.

Image Credit: Luca Volpi


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