Updated, April 3, 2013: A report of this meeting has been published in the journal, Neurology (2013;80:1161). This publication is an important step in creating standards for clinical trials of treatments in children with MS.
There are eight MS therapies that reduce MS activity, and even more under study – how can we safely study them in children who have MS? This was the focus of the “Emerging Therapies Summit,” a workshop convening representatives from drug regulatory agencies, MS advocacy organizations, pharmaceutical companies and pediatric MS experts, held by the International Pediatric MS Study Group (IPMSSG) and funded by the National MS Society and the MS International Federation. A report from the meeting will be published and a Clinical Trials Task Force is being created by the IPMSSG to drive consensus around designing and conducting trials in children.
Background: Although MS occurs most commonly in adults, it is also diagnosed in children and adolescents. Estimates suggest that 8,000-10,000 children (defined as up to 18 years old) in the United States have multiple sclerosis, and another 10,000-15,000 have experienced at least one symptom suggestive of MS. Increasing evidence suggests that the disease-modifying therapies are safe and well tolerated in children. However, clinical trials have not formally evaluated the safety or effectiveness of these therapies in children with MS.
Regulatory agencies are increasingly mandating that these trials are necessary when developing new therapeutic strategies for which pediatric use is anticipated. As a voluntary group of over 150 academic physicians and researchers treating and/or studying MS in children, the IPMSSG deemed the time right for a summit to consider the feasibility of trials in pediatric MS.
The Meeting: The IPMSSG amassed a capable group: More than 70 people from the academic and clinical pediatric MS field, representatives from three regulatory agencies (the U.S. Food and Drug Administration, the European Medicines Agency and Health Canada), members of MS patient organizations, and staff from seven pharmaceutical companies (both manufacturers of approved therapies and of experimental treatments in the MS pipeline) attended the summit.
The primary goal of the meeting was to analyze the feasibility, methodology, and priorities for pediatric clinical trials of new therapeutic strategies in MS. The group discussed current knowledge and best practices in several areas, including clinical outcome measures, clinical trial design, imaging outcomes, cognitive outcomes, pharmacology, drug safety and biological mechanisms, and drug safety registries.
Next steps: A comprehensive meeting report will be published in the near future, and a Clinical Trials Task Force is being created by the IPMSSG to drive consensus around the complicated process of designing and conducting trials in children. The goal is to ensure that advances in the treatment of MS in children are achieved in the shortest possible time frame with the highest degree of safety.
The National MS Society is a driving force in the effort to study MS in children, with the hope of stopping the disease in its tracks in the youngest people who have it, and the hope that understanding the disease in its earliest stages will help to end MS forever. Read more about pediatric MS and the network of Pediatric MS Centers of Excellence established by the Society.