A small group of children in Sheffield with the most severe forms of epilepsy will soon be among the first to be prescribed with cannabis in a trial that could pave the way for the treatment to be rolled out to hundreds more people.
In the next few weeks, Dr Chris Rittey, a consultant pediatric neurologist at Sheffield Children’s Hospital, has the unenviable task of identifying just five children from the hundreds on his books to act as trailblazers in a study of the drug’s effectiveness.
If deemed successful, cannabis-based treatments could then be made widely available as long as they are subsequently approved by the European Medicines Agency and National Institute for Health and Care Excellence (NICE), and are then funded by NHS England.
Dr Rittey said the study marks the latest stage of a global campaign that has taken years to come to fruition, based in part on long-held hunches among doctors going back to the 1990s.
These beliefs were later taken up in the United States by the parents of a girl called Charlotte Figi, whose social media campaign eventually led to the development of a drug now known as Charlotte’s Web.
“The medical fraternity had known for a long time that cannabis had an antiepileptic effect,” said Dr Rittey.
“But increasingly people started to say we need to investigate this properly.
“We needed to find out is this a really useful thing for people with epilepsy - one is it safe and two does it work.”
Dr Rittey said the particular part of the cannabis plant that the medical world is interested in terms of epilepsy is a compound called cannabidiol – or CBD.
He said that studies conducted last year into CBD’s affect on two of the most serious types of epilepsy that affect children - Dravet syndrome and Lennox-Gastaut syndrome - yielded positive results.
These conditions can cause children to have multiple seizures per day, often leading them to being repeatedly admitted to hospital or, in the case of Lennox-Gastau syndrome, having to wear helmets to protect them from so-called ‘drop attacks’.
“What those studies showed was that indeed CBD did work, but what it wasn’t was a magic bullet,” said Dr Rittey.
“What they tended to suggest was that about a quarter of people will have a significant benefit or see their seizures reduce by a quarter.
“So it is not like everybody is going to benefit but on the other hand you are talking about a group of people who have really awful epilepsy who have tried multiple other drugs, so if even 25 per cent of them get benefit that is worthwhile.
“You have to be realistic that it is probably not going to work for the majority of your patients but there will be some of them for whom it is going to make a huge difference.”
Although some trials have been done, CBD is not currently a licensed medication and is therefore not available to doctors to prescribe in the UK.
However, drug company GW Pharmaceuticals have offered an early access programme and made available 150 prescriptions to patients in the UK, 125 for children and 25 for adults, with five of the child prescriptions being available to Dr Rittey.
“Realistically there are going to be more than five children who could benefit from it so we have got to work out a way of identifying that five,” he said.
“In our area we know there are probably around 25 and I suspect we will be able to easily agree the top three or four.
“What will be really difficult is the fifth and we will have a whole group of children who are all equally deserving.”
“It will be a really difficult decision because there are going to be families who think their child would have benefited and they will be right.
“But it will be based on the evidence we have and which child is likely to get most benefit from it.”
CBD hit the headlines recently after the parents of a child treated at Sheffield Children’s Hospital complained that their twin boys were being denied treatment that could save their lives because the trust balked at paying the £30,000 it would cost per child, per year.
Dr Rittey, however, rejects this and says CBD has to go through the process of trial, licensing and commissioning like any other medication.
“There is a lot of hype about cannabis at the moment and some of that is real but the hype probably isn’t all that it is made out to be.
“I do an epilepsy clinic every Wednesday morning and a year ago maybe once every three or four clinics someone would ask me about CBD, three months ago someone would ask me about it every week. Now, every other patient asks me about it.
“I think at the end of this there will be a rationale that says this is who it is helpful for and doctors will have guidance about prescribing.
“But people who have medical condition that doesn’t respond to conventional treatments are always going to look for something else – and why wouldn’t you?”