Development of Novel Therapies for Pediatric Cancer

St. Jude Children'S Research Hospital, Memphis TN

Investigators

Abstract

DESCRIPTION (provided by applicant): Pediatric cancers are orphan malignancies that occur in patients who have historically been overlooked for the most part by the pharmaceutical and biotechnology industries; thus, the pediatric cancers can be considered double orphans" - orphan diseases that occur in orphan patients. Although dramatic strides have been achieved over the past 40 years in the management of the pediatric cancers, there remain many shortcomings in the efficacies and toxicities of available therapies for the various types of childhood malignancy. The major goal of this application is to facilitate the development of new technologies for the treatment, prevention, and diagnosis of pediatric cancer by leveraging the incentives provided through an AP4 Center to be based at St. Jude Children's Research Hospital. The secondary goal of this St. Jude AP4 Center - a goal critical to the successful creation and development of the Center, as well as its ability to ultimately become self-sustaining - will be to incorporate the activities of the Center within the context of a broader, regional effort involving academic, not-for-profit and for-profit organizations located primarily (but not exclusively) in the Southern U.S. that are committed to the development of a robust regional health care and biotechnology industry. To ensure the viability and success of the proposed St. Jude AP4 Center, we will utilize the input of numerous individuals from these organizations who have expertise in the clinical-translational and basic biomedical sciences, in fund-raising efforts from charitable, venture capital, governmental and other sources, in the ability to interact effectively with political figures, local and federal government agencies, in the protection and licensing of intellectual property, in the pie-clinical and clinical development of new therapeutic, preventative, and diagnostic technologies, and in marketing and commercialization strategies. The crucial involvement of these regional interests also grows in part out of the realization that it will likely remain difficult to identify partners from existing pharma and biotech for selected 'small market" technologies developed within this AP4 Center, even with AP4-sponsored incentives, necessitating the need for a well-developed infrastructure to foster the development of new for-profits to commercialize these novel methodologies for the better management of childhood cancer.