Lauren B. Krupp, MD: Kids with relapsing and remitting MS [multiple sclerosis] continue to have some unmet needs. There need to be more opportunities for support through things like telemedicine and telesupport groups, taking advantage of the fact that with new technologies we can bring kids together who are otherwise geographically dispersed. I think that it’s very hard for kids to have a condition that nobody else…among their friends, among their peers, or at their school has, and it is valuable for them to be able to share with others who have the same condition.
We need to have better ways of explaining to parents that their kids should be allowed to engage in sports and social activities. They need to be encouraged to feel like regular kids. We need more information on optimal diet. We know, already, that kids benefit from exercising, so we need to encourage parents and kids to engage in sports and exercise and be active because that’s healthy for the brain, the body, and the spirit. So those are some of the things.…Those people who have had relapses need the opportunity to get on more effective therapies. We need to come up with a way of repairing the damage that’s happened.
There’s a tremendous need for better biomarkers in pediatric MS as well as in adult MS. The need for biomarkers is not only to help determine which medication is the best for a particular person but also to tell us who’s really going to run into trouble. I saw a young child whose MS started when he was age 4. Now, at age 22, even though he’s on a very effective medicine, he has had so many relapses that he’s really not able to work. I saw another patient whose MS started when she was age 14. She was very noncompliant, to the point of not taking her medication at all. But I saw her again at age 31, and she’s doing great. So the medication is, of course, important, but it’s not the whole story. She had something in her nervous system that speaks to tremendous resilience. Whereas the other patient, despite going on one treatment after another, kept on relapsing and had tremendous consequences for those relapses.
So the first need for a biomarker is to determine who can recover from relapses and who cannot. Who will suffer neurodegeneration down the road, and who will not? Because the ones who are going to have degenerative changes—that’s the group for which you really have to put out all of your efforts to preserve what they have with the most effective medicines. You tell everybody to exercise and eat right, but that’s the group that really needs it. So that kind of biomarker is needed.
We also need biomarkers that will tell us which medication is most effective for this particular individual. We don’t have that. We do have some biomarkers that tell us this particular medication is associated with a risk of a particular infection. In the case of natalizumab, which is a highly effective medication, the biomarker for the antibody titer to the virus JCV [JC polyomavirus] is a good indicator of a person’s risk of having a serious adverse event with that medication. But we need similar biomarkers for the other agents and for other infections.
Brenda L. Banwell, MD: Pediatric relapsing MS remains a disease that is still a bit of an orphan. We have far less information on treatment effectiveness than is made available to our adult MS population. We recognize that the different treatments used for adults, while powerful, may be either more powerful in children, which could increase the risk of toxicity, or less effective in children, given that children have such a relapse rate. This is one of the main reasons why we advocate for carefully conducted clinical trials with endpoints that we can evaluate…to understand how effective a given therapy is, and, arguably as importantly or maybe even more importantly, determine what the short- and long-term safety implications are.
We need to understand the longer-term implications of having MS happen in childhood and the teenage years. We’ve learned a lot in the last decade about the clinical features and the MRI [magnetic resonance imaging] characteristics. There’s research that has looked into risk factors, and now we’re understanding therapies. But what we don’t really know is the long-term impact of pediatric MS on long-term quality of life; on vocational success; on how this disease impacts an individual’s future in terms of choices of partners and having a family; long-term fatigue; and long-term emotional health. All of this is important, and this will be a partnership between pediatric health care providers and our adult colleagues. Because our patients outgrow us, we need to work with our adult colleagues so that we can follow our patients throughout their lifetime and really understand whether all of the things we’re trying so hard to do when they’re children improve the quality of life as they become independent young adults.
So our biggest unmet need is that we need to understand our patients in the long term. I think that the rarity of pediatric onset MS necessitates not only a local team, but an international one. I will advocate for the International Pediatric Multiple Sclerosis Study Group, which is now over 170 individuals in over 44 countries, all of whom communicate on a regular basis to inform each other on care practice, educational activity, and resources for children and their families. The group also serves as a way to advocate with regulatory authorities for clinical trials that are well designed, that have meaningful outcomes, and that can be feasibly done. We will not move this field forward if we don’t do that.