A international trial has shown that a form of medicinal cannabis works to treat drug-resistant seizures in a rare yet very severe form of childhood epilepsy.
Australian families devastated by Dravet syndrome have new reason for hope, with researchers claiming there's scientific proof a cannabis extract can effectively treat the severe form of epilepsy.
A medicinal trial has shown cannabidiol (CBD) significantly reduces seizures in children with the rare genetic brain dysfunction and can even eliminate them altogether.
The New England Journal of Medicine reports five per cent of participants, or three patients out of 60, were seizure-free after 14 weeks.
"Now that's pretty exciting," says University of Melbourne Chair of Paediatric Neurology and Austin Health Director of Paediatrics, Professor Ingrid Scheffer.
Currently "nothing works" to relieve sufferers from potentially life-threatening seizures, however some anti-epileptic drugs can make a difference.
Prof Scheffer who was part of the study and runs a clinic dedicated to treating Dravet syndrome says the trial has shown CBD is just as good.
"Cannabis is made up of hundreds of compounds but there's one that's effective for epilepsy, we think, and this is the first proof that this is the case," said
Out of every 500 children with epilepsy, two at most are likely to have this form of the condition that starts in infancy and is marked by frequent and often prolonged seizures.
It is a complex childhood epilepsy and can result in sudden and unexpected death.
Almost all children with Dravet will develop an intellectual disability by age five, according to Prof Scheffer.
"It's a very frightening disease and has a higher mortality risk as well," she said.
An international team of researchers studied CBD in treating drug-resistant convulsive seizures in 120 children with Dravet syndrome across the US and Europe.
Cannabidiol is one of at least 113 active cannabinoids identified in cannabis.
The children were randomly assigned a CBD oral solution or a placebo in addition to standard anti-epileptic treatment.
After 14 weeks, the median frequency of convulsive seizures per month decreased from 12.4 to 5.9 with cannabidiol, compared with a decrease from 14.9 to 14.1 with placebo.
Nearly half recorded a 50 per cent drop in seizure frequency.
However side effects were more frequent in the cannabidiol group and included diarrhoea, vomiting, fatigue, increased body temperature, drowsiness and abnormal liver-function tests.
"It's not a benign drug, it's just like any of the other drugs that have side effects in the same way," noted Prof Scheffer.
For some patients CBD is going to be great but it won't cure all, she warned.
"The next question is whether cannabidiol is effective in other forms of epilepsy and it is great that there are trials already underway of cannabidiol in other groups of patients with epilepsy."