A MARIJUANA-based drug will be given to children in Scotland in the hope of transforming the treatment of severe epilepsy in one of the first trials of its kind.
Researchers at Edinburgh University and the Royal Hospital for Sick Children in the city are applying for approval to test an oil extracted from cannabis flowers on young people, including babies.
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Parents of children with extreme forms of epilepsy have reported the medicine, which has had almost all the component that causes a "high" removed, has dramatically improved the symptoms.
However, without robust clinical trials to investigate the safety of the drug there is concern parents desperate to alleviate the distressing condition could administer cannabis treatments without prescriptions or information about the potential risks.
In Scotland, around 54,000 people have epilepsy and in about one-third of cases existing treatments do not work. Some forms, such as Dravet Syndrome, can cause relentless seizures and serious physical and cognitive disabilities.
Dr Richard Chin, consultant paediatric neurologist at the Royal Hospital for Sick Children, Edinburgh, and director of the Muir Maxwell Epilepsy Centre at Edinburgh University, said: "The children can go from having a seizure every day to having even 200 seizures per day. This is what families have to go through. There is obviously a need to find alternative treatments."
It is a compound found in marijuana known as cannabidiol or CBD which has been found to help epilepsy sufferers. A pharmaceutical company has produced the oil which contains CBD but less than 1% of the psycho-active component, THC, from plants grown at a secret location in the UK. The drug is named Epidiolex.
Dr Chin said: "One of the exciting things about CBD isn't its seizure control alone - it is its anti-epilepsy properties. It also improves behaviour and cognition. You can put children in intensive care and stop seizures, but it does nothing for their quality of life, nothing for cognition and nothing for the families. To have a drug that stops the seizures and also has cognitive improvement is pretty amazing."
The first safety trial of the drug to receive approval from the Food and Drug Administration in the United States is under way in New York. So far, 25 children are involved and Professor Patricia Corby, associate director for research operations at New York University's Bluestone Centre for Clinical Research, described the findings so far as "extremely promising".
Dr Chin is applying for authority from both a research ethics committee and the Medicines and Healthcare products Regulatory Agency to launch a similar trial in the UK led from Edinburgh, but also involving Great Ormond Street Hospital, London.
He hopes to receive approval before the end of the year and intends to recruit 30 children at each hospital to test the drug. Children will be given small doses of the oil twice a day.
Ann Maxwell, who founded the Muir Maxwell Trust in Scotland to help those affected by epilepsy after caring for her youngest son Muir who suffers Dravet Syndrome, has been instrumental in bringing together Dr Chin and Professor Helen Cross, epilepsy expert at Great Ormond Street Hospital, with the New York researchers - helping drive forward plans for a UK university trial.
Mrs Maxwell, of Midlothian, said: "Muir has in an awful lot of ways had his life taken from him by epilepsy as have we (as a family) to a certain extent. If I could wave a magic wand and give him his life back, I would. I love the idea we can take the damaged brain and reverse it. If that is possible there is still hope for Muir."
The Muir Maxwell Trust is seeking to raise £250,000 to support the trials of Epidiolex and is looking for donors. A debate about tackling the stigma of epilepsy through education takes place in the Scottish Parliament tomorrow.