The trial involved 215 young people aged between ten and 17 from around the world, with Great Ormond Street Hospital (GOSH) coordinating the UK arm of the study and treating several patients at the National Institute for Health Research (NIHR) GOSH Clinical Research Facility. Young people on the trial received a weekly injection plus a daily tablet - one was a dummy drug and the other was the real drug, fingolimod.
Dr Cheryl Hemingway, Consultant Paediatric Neurologist at GOSH said, “The data on Gilenya [the trade name for fingolimod] in this group of patients are some of the most promising we have ever seen. These young people could have more time to live their lives free of significant, disabling symptoms. Our hope now is that this will enable us to fundamentally change the course of this disease earlier than ever before”.
MS, which affects an estimated 10,000 children worldwide, is a disorder of the immune system where the layer that surrounds and protects the nerves in the brain and spinal cord is damaged. Young people with MS often experience relapses - spontaneous attacks of symptoms such as blurred vision, mobility issues, and bladder and bowel problems.
The results showed that children given the fingolimod pill had an 82 per cent lower relapse rate compared to those given the weekly injection of interferon - the current standard treatment for MS. The tablet, which is taken orally and therefore much more convenient for patients, also led to a reduction in the number of brain lesions and less brain shrinkage. Given the positive results, all young people who took part in the trial are continuing to take the fingolimod tablet and being closely monitored as part of the trial extension study.
Fingolimod, which is also known by the trade name Gilenya is already licenced for use by adults with MS, where it has shown similar positive results. Now, Novartis, the pharmaceutical company which makes the drug, is applying for a licence to prescribe it to children.
The research was presented at an MS research conference in Paris in November.
Isabel, 15, from Hertfordshire was diagnosed with MS at GOSH in November 2015. She took part in a clinical trial for fingolimod.
“It all started with a numb tingly feeling in my hand which recovered fully after about four months. A year later half of my face had dropped, so when I laughed or smiled, only half of my face would lift. I also at one point had double vision, which lasted a week. It was a bit embarrassing as I was laughing with only half a smile. The hand symptom was quite annoying as it took extra time and work to be able to copy notes and write from the board at school.
“I was referred to a consultant and from there onto GOSH, where after going through some difficult tests, it was confirmed that I had MS. I was quite stunned. I had known people with MS and seen various news features about it online, but never even knew that children could be diagnosed with it.
It took over a year to be diagnosed as it was initially thought to be nerve inflammation. Although the process was actually very quick once I was referred to GOSH.
“On the drug trial I had one injection per week and a tablet every day. One was a placebo (dummy drug) and one was real and neither me, nor Dr Hemingway knew which one was which. This drug had only been tested on adults and I was in the trial seeing if this drug was safe for other children to use.
“The drug has been great. It has made my life a lot easier and I would say that I am now happier all in all, as I am quite confident that I won’t have another relapse, which is the most life-changing thing. The drug supresses the white blood cells that attack my nerves but which also allow my immune system to work effectively. This makes me more prone to infection as my immune system is a bit weaker than most. So far though, I have only really had a cold. The drug is working for me and I hope it can continue to work for others with MS as it would be a major breakthrough.”
Read more about pioneering clinical research at GOSH