Pediatric researchers, investigating the biology of brain tumors in children, are finding that crucial differences in how the same gene is mutated may call for different treatments. A new study offers glimpses into how scientists will be using the ongoing flood of gene-sequencing data to customize treatments based on very specific mutations in a child's tumor.
"By better understanding the basic biology of these tumors, such as how particular mutations in the same gene may respond differently to targeted drugs, we are moving closer to personalized medicine for children with cancer," said the study's first author, Angela J. Sievert, M.D., M.P.H., an oncologist in the Cancer Center at The Children's Hospital of Philadelphia.
Sievert, working with co-first author Shih-Shan Lang, M.D., in the translational laboratory of neurosurgeon Phillip Storm, M.D., and Adam Resnick, Ph.D., published a study ahead of print today in the Proceedings of the National Academy of Sciences.
The study, performed in cell cultures and animals, focused on a type of astrocytoma, the most common type of brain tumor in children. When surgeons can fully remove an astrocytoma (also called a low-grade glioma), a child can be cured. However, many astrocytomas are too widespread or in too delicate a site to be safely removed. Others may recur. So pediatric oncologists have been seeking better options---ideally, a drug that can selectively and definitively kill the tumor with low toxicity to healthy tissue.
The current study focuses on mutations in the BRAF gene, one of the most commonly mutated genes in human cancers. Because the same gene is also mutated in certain adult cancers, such as melanoma, the pediatric researchers were able to make use of recently developed drugs, BRAF inhibitors, which were already being tested with some success against melanoma in adults.
The current study provides another example of the complexity of cancer: in the
|Contact: John Ascenzi|
Children's Hospital of Philadelphia