Single-cell Profiling of Pediatric Bone Sarcomas
Lay Summary: Each year in the U.S., about 1600 children and young adults are diagnosed with sarcoma--tumors affecting bones, muscle or cartilage. The majority of these are bone tumors, which include osteosarcoma (800 children diagnosed each year) and some Ewing sarcomas (250 cases each year). Bone sarcomas are rare, heterogeneous, and not yet fully characterized. Survival rates are in the 60% and as low as 20-30% for metastatic disease, and outcomes have not improved much over the past decade. To find novel effective approaches for sarcoma therapy, we need to better understand it at a single cell and molecular level. Novel techniques now allow us to get a "snapshot" of each and every single cell individually. These snapshots give us detailed information about which genes are expressed in any and every cell. By comparing these gene expression profiles, we can identify cells that are similar or different within the same tumor.
Lay Summary Project Goal: The first goal of our project is to contribute to the Single-cell Pediatric Cancer Atlas. We will do so by generating single-cell snapshots from 3000 cells for each of 35 pediatric bone tumors in total. We will also generate individualized avatar models for 10 of these cases and test the sensitivity to 500 drugs. The gene expression data will be used to determine cell subpopulations and will be correlated to clinical information as well as to drug sensitivity data. Our goal is to find if different proportions of cells correlate, for example, with treatment failure, disease recurrence or drug sensitivity, with the overarching goal of identifying novel therapeutic avenues for pediatric bone tumors.