Researcher:  Elizabeth A. Beierle, M.D.(Principal Investigator); Jamie M. Aye, M.D. (Co-Investigator) 

Project Title: “Development, Maintenance and Utilization of a Pediatric Solid Tumor Xenograft Bank”  

Hospital Affiliation: Children’s Hospital of Alabama 
Location: Birmingham, AL 
Year Funded: 2019 & 2020 

Summary: 

Despite progress in pediatric cancer care, the treatment for many pediatric solid tumors has not significantly changed in the past twenty years. Children with relapsed or resistant disease have even fewer treatment options available and almost none that result in cure. It is clear that these children will require novel and innovative therapies that target their disease.  

This research grant will enrich the pediatric solid tumor xenograft program that has been established and evaluate novel therapeutics for pediatric solid tumor types that are traditionally understudied and underfunded. 

Project Details: 

Despite progress in pediatric cancer care, the treatment for many pediatric solid tumors has not significantly changed in the past twenty years. Children with relapsed or resistant disease have even fewer treatment options available and almost none that result in cure. It is clear that these children will require novel and innovative therapies that target their disease.  

The development of new therapies, including the repurposing of approved drugs, requires significant pre-clinical testing prior to advancement to use in the clinical arena. Current models often evaluate new therapies utilizing long-term passaged human tumor cell lines. These cell lines may not always recapitulate the human condition from which they were derived. Research has demonstrated that human primary tumors directly implanted into immunodeficient mice more accurately recapitulate the features of patient tumors compared to cell based models. The use of primary human patient tumor xenografts has been well established in the study of human brain tumors and adult solid tumors. However, these models are not well established or universally available for the investigation of pediatric solid tumors. We have utilized internal funds to initiate a pediatric solid tumor tissue bank, thus demonstrating the feasibility of the proposed project. We have also completed a number of preliminary studies utilizing tumors from this xenograft bank, demonstrating the tremendous potential of this resource.  

The goal for the proposed project is to greatly expand this tissue bank and provide an avenue for the investigation of novel compounds for these tumors. Further, we hypothesize that the response of tumors to many novel compounds is related to tumor “-omics”, and proposed to investigate the molecular characteristics of the tumors that potentially contribute to specific therapeutic responses. The long-term goal is to identify agents that are effective treatments for children with tumors having specific genetic and molecular profiles, and move those agents more rapidly into the clinical realm.