Development of Novel Therapy for Childhood Cancer: Promise and Challenges

Only a limited number of phase 1 and phase 2 trials of new agents can be undertaken in the childhood cancer population. The Pediatric Preclinical Testing Program (PPTP) is an experiment to determine whether using xenografts derived from childhood cancers can be used to identify agents, or combinations of agents that may have significant clinical activity. Thirty one drugs or biological agents were tested against the PPTP in vivo panel. Tumors were characterized by expression and SNP profiling. By expression profiling xenografts clustered with histotype and their clinical counterpart. By SNP analysis gains and lo…