Pediatric Multiple Sclerosis: A Game-Changing New Therapy

Brenda L. Banwell, MD


September 14, 2018

Editorial Collaboration

Medscape &

Good morning. My name's Brenda Banwell. I'm the chief of neurology at the Children's Hospital of Philadelphia. I serve as the director of the Pediatric Multiple Sclerosis Clinic at CHOP. I recently served as one of the co–principal investigators of the first-ever pediatric multiple sclerosis (MS) clinical trial, a trial that compared fingolimod, or Gilenya®, to interferon beta-1a by a subcutaneous injection.[1] Before we discuss the trial, I want to start by briefly reviewing a little bit about treatment of MS in children.

Pediatric-onset MS is a relapsing-remitting disease. Patients experience clinical relapses [which are visible on] MRI as accrual of new focal lesions. Most of the therapies that have been used in children to date have targeted this inflammatory biology in relapsing MS. For the past 15-17 years, treatment of adults with relapsing-remitting disease has focused on a variety of compounds, none of which, until recently, had ever been formally studied in children. As a consequence of that, we have used off-label medications aimed at reducing relapse frequency and limiting the acquisition of new [lesions detectable] by MRI visualization.

This recently completed trial, and the approval by the US Food and Drug Administration (FDA) of Gilenya, represents a major step forward in the care of children with MS. We now have evidence-based guidelines for the use of oral fingolimod for pediatric-onset MS. The study showed that pediatric patients with relapsing-remitting MS treated with this agent had an 82% reduction in relapse rate compared with those receiving interferon beta-1a.

Furthermore, there was a marked decrease in the number of new MRI lesions in the fingolimod-treated patients compared with those treated with interferon beta-1a. Excitingly, the rate of brain volume loss in treated patients was also reduced. All of these outcomes suggest that treatment of pediatric-onset MS early in the disease course has a major benefit for those children and youth offered this opportunity.

At this point, the pediatric MS landscape is expanding. Not only do we now have access to an FDA-approved therapy, but we also have multiple clinical trials ongoing. For every new treatment for adult-onset MS, we must give consideration to whether a pediatric trial can or should be done. As a consequence, many of the exciting advances in care are being brought forward at an evidence-based level for pediatric-onset MS patients.

My ending message is that the landscape and the future of the treatment of MS in childhood are far brighter than they were 15 or 20 years ago.

Of note is the fact that we have also now had 15 years of experience using some of the therapies that have become available during that timeframe. Interferon beta-1a and its various formulations and glatiramer acetate (Copaxone®) have been our first-line therapies in the treatment of pediatric-onset MS for many years now. These therapies have benefit for some of our patients and certainly now have 15 years of favorable safety profiles. These data has been reviewed in various retrospective publications.[2,3,4] There have also been some retrospective reviews of treatments like natalizumab (Tysabri®).[5,6]

Therefore, although Gilenya has emerged as the first-ever formally approved treatment, that does not mean it's the only option for children with relapsing-remitting MS. There are [other] highly effective therapies that are often offered to children with very rapidly progressing relapse frequencies or children whose MRIs show a very high burden of disease.

We are excited about the potential for therapies that act on B-cell biology, such as ocrelizumab or its cousin therapy, rituximab, as these treatments have been shown to be extremely efficacious in adults.

My ending message is that the landscape and the future of the treatment of MS in childhood are far brighter than they were 15 or 20 years ago. I am excited to see that we are moving forward with evidence-based therapy for this very needy pediatric population.

I thank all of you for your kind attention, and I hope that you also have access—not only to different treatments, but to regional pediatric MS centers that can provide expertise and surveillance for safety, which remain very key aspects of the care of every child with MS. Thank you.