Diagnosis and Management of Multiple Sclerosis in Pediatric Patients - Episode 10

Drugs and Biomarkers in Development for Pediatric MS


Expert neurologists discuss drugs in development for pediatric multiple sclerosis and consider the potential for biomarkers to monitor treatment response or new disease activity in the future.

Lauren B. Krupp, MD: The medications used in adults with MS [multiple sclerosis] are being examined for pediatric MS in clinical trials. There have been clinical trials done with teriflunomide and dimethyl fumarate; there have been clinical trials done with the infusions or being planned with the infusions, specifically with ocrelizumab. There are studies that are planned with other oral S1P [sphingosine-1-phosphate] modulators, such as siponimod or ozanimod. There are pediatric MS studies being planned for almost all of the therapies that are used in adults with MS.

Tanuja Chitnis, MD: It’s a very exciting time for pediatric MS. There are a number of therapies in development or in clinical trials for this disease. There has been a recent clinical trial of Aubagio [teriflunomide], which has just been completed. There are several clinical trials of dimethyl fumarate that are ongoing, and we should get some results from those soon. There’s also a new category of therapy that has been used extensively in adult MS, which is now being trialed in pediatric MS. These include studies for ocrelizumab as well as for ofatumumab, that will be launched. There has been a fair bit of off-label use of a cousin of those 2 drugs called rituximab, and there have been open-label studies and case series evaluating rituximab as a treatment for pediatric MS.

As I mentioned earlier, it’s very important to monitor patients regularly and to ensure that they are not having either new clinical relapses or new MRI lesions. There’s now an evolving role for biomarkers to monitor disease course both in adults and potentially in children with MS; this includes a biomarker called neurofilament light chain, which can be measured in the serum and is very accessible. We might see this or other biomarkers come to the foreground as a way to monitor treatment response and to monitor for new relapses or disease activity. That’s very exciting because then we’ll be able to really track how individual patients are doing, and ensure they are on the right therapy and be able to switch as early as possible.

Transcript Edited for Clarity