Drug found to have 'very significant benefit' for children and teenagers with multiple sclerosis
A drug which is used to treat multiple sclerosis in adults will now be recommended for treatment in children and teenagers with the condition.
Prof Orla Hardiman, consultant neurologist at Beaumont Hospital and Director of Trinity Biomedical Sciences Institute presented recent findings of a study to international doctors to show that the drug fingolimod is effective for MS patients under-18.
Fingolimod is currently used for adult patients who have relapse-remitting MS.
“Paediatric MS patients experience more frequent relapses and are more likely to accumulate physical disability at an earlier age than those diagnosed as adults,” said Professor Hardiman.
And the study signified “an important step towards a potential new treatment that could improve the lives of these young patients”, she said.
Professor Hardiman presented the information at the European and Americas Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS-ACTRIMS) meeting last Saturday.
Currently there are no specifically approved disease modifying therapies for children and adolescents with MS, a population at high risk of long-term disability.
Multiple Sclerosis is a progressive, neurological condition of the brain and spinal cord (central nervous system). It can be a highly debilitating disease which touches every aspect of young patients’ daily lives, from school performance to family relations and friendships.
“MS is really a disorder of adulthood but we do have some children who present with paediatric MS and they come to us, and once they hit 18 they go to the adult service,” Professor Hardiman said.
“Obviously the earlier we get treatment for people the better, particularly for children, it can stop the course of the disease.”
Fingolimod is not currently approved for the treatment of paediatric MS.
The study found that those treated with fingolimod had significantly less brain shrinkage (measured by MRI as brain volume loss), compared to those treated with interferon beta-1a1. Brain shrinkage in adults is associated with the loss of physical and cognitive function.
“The drug is licensed in adults, but it wasn’t licenced in children. What happened last week, the results of the study showed categorically a very significant benefit for children. The data are very robust,” Professor Hardiman said.
“In terms of its availability and utility, it’s a very easy-to-use drug… The hope would be that the licensing would be extended to children under-18.”
Novartis, the pharmaceutical company, says it is working on a submission with health authorities worldwide.
Dr Simon Schmid, Chief Scientific Officer at Novartis Ireland, said “This pioneering study demonstrates our continued commitment to providing new treatment options for people with MS with the highest need. We look forward to working with health authorities to make this treatment available for people with MS.”