Trials are finally starting into the benefits of cannabis-based medicine for children and young people with epilepsy after a mum spent years campaigning.
Charlotte Caldwell’s son Billy used to have up to 300 seizures a day until he started on medicinal cannabis.
Doctors have so far been reluctant to prescribe cannabis products on the in the absence of clinical trials, even though they are legal.
This has led a booming market where private doctors are able to charge desperate parents up to £2,000 a month.
Charlotte started her crusade six years ago after Billy’s medicinal cannabis, prescribed by a doctor in Canada, was seized at Heathrow Airport. That led to the law change and Billy, 18, getting his NHS funded prescription cannabis in 2018.
But the lack of formal trials means thousands of other parents still have to go private.
Charlotte hopes the major breakthrough with the trials could make cannabis-based medicines more readily available on the NHS.
She said: “If the trials are successful, it means no other child suffering with refractory epilepsy will have to endure the long and tortuous journey Billy did to receive life-saving treatment on the NHS.”
Two trials will take place, run by University College London and funded by the National Institute for Healthcare Research, with 500 NHS patients who meet criteria.
Charlotte, of Castlederg, Northern Ireland, said: “The news is historic. It’s amazing. I’ve been campaigning for this to happen for years.”
The first proposed trial will investigate cannabis-based medicines for adults and children with epilepsies that start in the first three years of life. The second will look at patients with genetic generalised epilepsies who have not responded to standard treatment.
Researchers from UCL and Great Ormond Street Hospital will study whether the medicines are safe and effective in reducing seizures, as well as whether they affect learning, sleep, behaviour, quality of life, stress and anxiety.
Data could in future be used to enable products to be licensed by medicines regulator the MHRA and approved by the NHS, if they are both clinically and cost effective.
Thousands of children and adults suffer from uncontrolled epilepsy.
One of those likely to benefit if the trials are successful is Harry Siddans, 17, from Great Dunmow, Essex.
His mum, Marie, said: “The trials will be a game changer. It means we will know that prescription cannabis is manufactured safely and to a standard like any other drug.
“Getting supplies can be difficult, you can never be sure the drug you are getting is the same one month to the next.”
Harry has severe refractory epilepsy that cannot be controlled by anti-epileptic drugs.
He was having up to a dozen seizures a day, some life-threatening. The family started using prescription cannabis in 2022 and it cut seizures to one or two a week. Before that, he could not walk or go to school.
But his treatment comes with a monthly bill of £995 for Marie and her gardener husband, William.
The couple have spent more than £30,000, borrowing from family to cover the cost. Marie said: “When it’s your child, you’ll do anything.”
Figures show fewer than five NHS prescriptions for unlicensed prescription cannabis were given from November 2018 to July 2022.
Co-chief investigator Professor Helen Cross, of UCL, said: “This is an essential step on the pathway to establishing whether these medicines are safe and effective.”
Consultant paediatrician Dr Jo Jones, who has a daughter with epilepsy, said: “It’s fantastic that we are having these trials. Thousands of patients can benefit.” Young Epilepsy, which works with children and young people with epilepsy, said: “Measuring the impact of cannabis-based medicines on the lives of children and young people with epilepsy will help clinicians better understand the benefits and risks.
“We hope this can go towards improving the lives of children and young people living with drug-resistant epilepsy.”
