Dr. Evans and others convened a group of doctors and scientists to create a scientific plan that would clearly outline what needed to be done to develop new drugs for pediatric cancers. The effort involved partnerships with academia, large and small pharmaceutical companies, government health agencies, and philanthropic foundations.

St. Jude Children’s Research Hospital has focused on filling the gap in the discovery of pediatric cancer drugs by building a Good Manufacturing Practices (GMP) facility, and launching a Chemical Biology and Therapeutics initiative. There is interest in leveraging these efforts through the National Cancer Institute’s (NCI’s) Pediatric Cancer Drug Discovery Consortium under an expanded public–private–government initiative. The GMP facility is producing gene therapy vectors, vaccines, and monoclonal antibodies. The Chemical Biology and Therapeutics initiative will involve putting viable targets in pediatric tumors from the research laboratories of St. Jude’s and others through high-throughput screens of very large libraries of small molecules—currently more than 1 million compounds. This is similar to the type of drug screening a pharmaceutical company might conduct, although St. Jude’s has objectives other than simply developing new drugs. The goals are to identify small molecules that are inhibitors of specific targets in pediatric cancers and could be used to explore the pathways involved in pediatric cancers, identify candidate small molecules for preclinical testing, and network with others to improve capacity and ultimately advance these agents to the level of the pediatric clinic. Finally, NCI is funding the Pediatric Cancer Drug Discovery Consortium to screen adult cancer drug candidates in animal models with pediatric tumors.

In response to Dr. Evans’ presentation, Dr. Nelson raised concerns about relying on independent, well-funded pediatric institutions to compensate for the deficits in funding for traditional academic research. Dr. Evans replied that all involved need to do what they can, and that he is concerned about the flat funding for the National Institutes of Health. To address the funding issue, he plans to rely more on team science, looking to academic partners and others for what they do well.

Dr. Susan Weiner, a member of the audience from the Children’s Cause for Cancer Advocacy, asked whether St. Jude’s expects to file a New Drug Application if an entity is found that is effective in only a small group of children. Dr. Evans replied in the affirmative and noted that St. Jude’s has acquired various libraries of compounds through different arrangements. Some have been purchased outright; others involve the right to first refusal if a lead compound or potential therapeutic is found. Dr. Evans warned that, with molecular characterization of tumors, more and more drugs will be found to be useful in smaller subsets of patients. A new business model needs to be devised for the development of these drugs with small potential markets, a need that is growing for adult diseases as well.

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