The National Cancer Institute (NCI), part of the National Institutes of Health (NIH), recently awarded a five-year grant to a research team at Baylor College of Medicine in Houston, Texas, to join the Pediatric Preclinical Testing Consortium, whose goal is to address key challenges associated with the development of novel therapies for children with cancer.
The Baylor research team will focus their efforts on brain cancer, the primary cause of death related to cancer in children. The team will be led by Baylor’s associate professor of pediatrics Dr. Xiao-Nan Li, who is also a member of the Texas Children’s Cancer Center.
One of the important drug development challenges to be tackled by the Consortium is the generation of reliable data from studies with laboratory and animal models that enable scientists to define which therapeutic agents are worth pursuing in human clinical trials.
“One of the challenges in clinical drug development is how to effectively prioritize drug candidates to ensure clinical success in cancer patients,” explained Dr. Li in a press release. “As pediatric drug candidates are much more numerous than the small number of patients, it is essential to perform comprehensive preclinical testing to identify the investigational agents that are most likely to be effective in the clinic. However, such effort has been blocked for many years due to the lack of clinically relevant and molecularly accurate model systems.”
Dr. Li is a recognized leading researcher in the field of pediatric brain tumor model development and drug testing in vivo. He developed a pioneering surgical technique to implant, in a rapid and safe manner, human brain cancer cells into several locations (cerebellum, cerebrum and brain stem) in the brains of mice. These animal models mimic human brain cancer and enable scientists to study in more detail the mechanisms underlying the disease and to assess novel therapeutic agents.
Within this project, both in vitro and in vivo experiments will be conducted in order to achieve two main goals: to shed light over the mechanisms of cell killing and the causes underlying the development of therapy resistance, and to assess the therapeutic efficacy of potential agents in appropriate genetic models in terms of survival and alterations in tumor regression or progression.
“Our objective is to make use of our unique panel of animal models to examine therapeutic efficacy of new agents and to analyze mechanisms of action and therapy resistance in high-grade glioma, medulloblastoma and ependymoma. Our hypothesis is that these patient-specific model tumors will respond to anti-cancer therapies similarly to the corresponding human primary tumors, and the effective agents identified through this system would have better chances of clinical success.” concluded Dr. Li.
Besides the Baylor research team, NCI awarded similar grants to join the Pediatric Preclinical Testing Consortium to four more groups involving other institutions.