A marijuana plant
A marijuana plant

New cannabis-based epilepsy drug showing 'promise' in clinical trials on children

Epidiolex was developed for children with illnesses that resist other treatment

Lizzie Dearden
Wednesday 15 October 2014 16:55
Comments

A new cannabis-based drug for children with severe epilepsy is showing “promising” signs in clinical trials.

Epidiolex is being tested on children with Dravet Syndrome and other forms of epilepsy that do not respond to existing drugs.

Its manufacturer, GW Pharmaceuticals, said most of the 60 children in the trials so far had seen the frequency of both “drop” and convulsive seizures fall.

The most common side effects were somnolence (sleepiness) and fatigue in a fifth and a tenth of patients respectively.

Dr Elizabeth Thiele, director of the Pediatric Epilepsy Program at Massachusetts General Hospital, said she was “very encouraged” by the preliminary results.

“I believe that Epidiolex has the potential to be an important advance in treatment for these treatment-resistant children and will likely have a significant role as a future therapy,” she added.

The drug, which does not have any intoxicating effects, is a liquid made of a purified cannabidiol extracted from marijuana plants grown under licence at a secret location in Britain.

The US Food and Drug Administration fast-tracked the drug’s status to allow the trials earlier this year and placebo-controlled clinical trials for Dravet Syndrome and Lennox-Gastaut Syndrome are due to start.

Both forms of epilepsy start in childhood and are difficult to treat.

Out of 151 patients in safety testing for the drug, 26 experienced serious adverse effects and two died, although GW Pharmaceuticals said independent investigations found they were unrelated to the trial.

The children in the study, with an average age of 11, were all receiving other treatment alongside the Epidiolex and two were removed from the study because of “adverse effects” and another four due to “a lack of clinical effect”.

The drug trials could soon begin at Great Ormond Street

The patients are all between the ages of one and 18 with illnesses shown to be resistant to many or all of the anti-epilepsy treatments, including drugs and a ketogenic diets.

Maria Roberta Cilio, who is testing the drug at the Benioff Children’s Hospital in San Francisco, said studies on animals had showed cannabidiol works as an anticonvulsant.

“It’s important to get seizure control at any age but in children, uncontrolled seizures may impact brain and neurocognitive development, which can have an extraordinary effect on quality of life and contribute to progressive cognitive impairment,” she added.

“This trial is pioneering a new treatment for children with the most severe epilepsies, for whom nothing else works.

“But we are just at the beginning, combining experts in the field with a brave institution ready to take this on, and courageous patients looking for hope.”

Epidiolex has only been tested on American children so far but trials are expected to expand across Europe, including at Great Ormond Street Hospital in London.

Register for free to continue reading

Registration is a free and easy way to support our truly independent journalism

By registering, you will also enjoy limited access to Premium articles, exclusive newsletters, commenting, and virtual events with our leading journalists

Please enter a valid email
Please enter a valid email
Must be at least 6 characters, include an upper and lower case character and a number
Must be at least 6 characters, include an upper and lower case character and a number
Must be at least 6 characters, include an upper and lower case character and a number
Please enter your first name
Special characters aren’t allowed
Please enter a name between 1 and 40 characters
Please enter your last name
Special characters aren’t allowed
Please enter a name between 1 and 40 characters
You must be over 18 years old to register
You must be over 18 years old to register
Opt-out-policy
You can opt-out at any time by signing in to your account to manage your preferences. Each email has a link to unsubscribe.

By clicking ‘Create my account’ you confirm that your data has been entered correctly and you have read and agree to our Terms of use, Cookie policy and Privacy notice.

This site is protected by reCAPTCHA and the Google Privacy policy and Terms of service apply.

Already have an account? sign in

By clicking ‘Register’ you confirm that your data has been entered correctly and you have read and agree to our Terms of use, Cookie policy and Privacy notice.

This site is protected by reCAPTCHA and the Google Privacy policy and Terms of service apply.

Register for free to continue reading

Registration is a free and easy way to support our truly independent journalism

By registering, you will also enjoy limited access to Premium articles, exclusive newsletters, commenting, and virtual events with our leading journalists

Already have an account? sign in

By clicking ‘Register’ you confirm that your data has been entered correctly and you have read and agree to our Terms of use, Cookie policy and Privacy notice.

This site is protected by reCAPTCHA and the Google Privacy policy and Terms of service apply.

Join our new commenting forum

Join thought-provoking conversations, follow other Independent readers and see their replies

Comments

Thank you for registering

Please refresh the page or navigate to another page on the site to be automatically logged inPlease refresh your browser to be logged in