Description
On September 14-15, 2011, the NHLBI sponsored a workshop through the Production Assistance for Cellular Therapies (PACT) program. The goal of this workshop was to identify the unique opportunities and challenges of developing cellular therapies in the pediatric population and to identify potential strategies to facilitate the development of new, effective cellular therapies in diseases primarily affecting children. The diseases and conditions affecting pediatric patients for which cellular therapeutics may offer the only potential curative treatment at this time are almost exclusively rare diseases. The ethical considerations of conducting clinical trials in pediatric patients combined with challenges of conducting trials involving cellular therapy for rare disorders were considered in the workshop. The speakers, panelists and participants identified the unmet needs delaying the advancement of clinical research for these childhood conditions and proposed strategies to ameliorate the obstacles hindering the development of novel therapeutics.