The 215 children, aged 10 to 17, were given the drug fingolimod, brand name Gilenya, as part of an international clinical trial, against a backdrop of “significant unmet need” for safe and effective MS treatments for children and adolescents.
Órla Hardiman, one of the country’s leading consultant neurologists, said the trial results were “good, really good”, for children with relapsing MS.
While Prof Hardiman is an adult neurologist at Beaumont Hospital, children with the disease are co-managed by paediatric and adult neurology services, transferring over to the adult service at age 17.
Prof Hardiman said that children with relapsing MS currently receive weekly intramuscular injections of interferon “and kids don’t like injections”. A lot of them relapse, leading to disability “at a pivotal state in life”.
She said there had been discussions over the years with Novartis, the manufacturer of Gilenya, about the possibility of children taking oral therapeutics but the clinical data wasn’t there to support that approach.
“We did not have the data to say if fingolimod was safe or efficacious in children. But this trial [Paradigms] asked a very specific question and it got a very specific answer,” Prof Hardiman said.
“It provides Class One evidence that this drug should be prescribed for kids.”
Paradigms, the first controlled, randomised trial specifically designed for paediatric MS, also found that children treated with fingolimod had significantly fewer brain lesions than those on interferon injections.
Fingolimod, described as “disease-modifying therapy”, is not currently approved for the treatment of paediatric MS but Prof Hardiman said she imagines that, over time, and given the “robust clinical data”, the Health Products Regulatory Authority will approve it. She said it would not be a huge cost to the exchequer due to the small number of children with MS. It is already available to adults under the Hi-Tech Drugs Scheme.
Prof Hardiman said paediatric MS patients experience more frequent relapses and are more likely to accumulate physical disability at an earlier age than those diagnosed as adults
There are 9,000 people with MS in Ireland of whom between 3%-5% are estimated to be children.