Programs and Research
Investigators in the Pediatric Cancer and Leukemia Program investigate childhood malignancies of the nervous system (brain tumors, neuroblastoma), the eye (retinoblastoma) muscle and bone (sarcomas), and blood cells (leukemias). The following highlights some of CHLA’s research accomplishments.
Gene Activity Profile Identifies High-Risk Neuroblastoma. Neuroblastoma, a tumor of the sympathetic nervous system, is one of the most common solid tumors in infants and children. Using “gene chips” Drs. Shahab Asgharzadeh and Robert Seeger discovered 55 genes whose (activity) expression can be used to determine the chance of survival for children with widespread (metastatic) disease. This information also likely will lead to the discovery of genes and networks of genes that could be targeted in new therapeutic strategies. Collectively, this research will make it possible to individualize care for each patient based on definition of a tumor’s gene signature.
Normal cells can form blood vessels that help cancers to grow. Cancer cells must survive and grow in their microenvironment in order to become a problem for patients. Drs. Anat Epstein and Yves DeClerck have provided new information about how blood vessels, which are essential for providing nutrients and oxygen to the tumor cells, form. Dr. Epstein found that cells lining blood vessels must have an anchoring foundation in order to survive and maintain blood flow. Dr. DeClerck showed that cells forming a supporting scaffold around these lining cells also are key to maintaining a nutritious blood supply to tumors. Both of these cell types can be targeted therapeutically to disrupt blood supply and thus to cause tumors to stop growing or shrink.
Normal cells can help leukemia cells resist treatment. Drs. John Groffen and Nora Heisterkamp used a model of childhood leukemia that they developed in mice to understand how resistance to the drug imatinib (Gleevec) may develop in patients. They showed that normal cells could protect leukemia cells from being killed by this drug. This is important because it suggests that imatinib may be more effective in children if this effect of normal cells could be blocked.
Integrated research teams continue to thrive. CHLA uses “bench to bedside” translational research strategy for neuroblastoma to continue developing teams of laboratory and clinical investigators who work together. Failure of current therapies for brain tumors and leukemias are among the greatest challenges facing us. Thus, funding from Bogart is providing “seed capital” to establish outstanding groups of laboratory and clinical investigators to tackle these two problems. For example, studies of young children with brain tumors seek to cure them and importantly to also preserve their ability to learn and function in society. Thus, CHLA's brain tumor researchers seek to “save lives and save neurons”.
Clinical testing of new treatments. In the area of clinical trials, Bogart physicians test new therapies that are based upon laboratory findings. We have organized multi-institution consortia that specialize in testing new strategies for childhood neuroblastoma, brain tumors, and leukemia. CHLA is the only institution in the US that leads three clinical trials consortia.
Read more about this topic: Bogart Pediatric Cancer Research Program
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