Prospective Validation of a Prognostic Liquid Biopsy Approach for Pediatric Ewing Sarcoma
Dr. Brian Crompton is a physician-scientist at Dana-Farber Cancer Institute and his research focuses on finding new treatment approaches for patients with Ewing sarcoma. Ewing sarcoma is an often devastating pediatric cancer of the bone whose only effective treatment employs intensive chemotherapy, surgery, and radiation.
The treatment for aggressive pediatric sarcomas has remained largely unchanged for the past 15 years and improvements in outcomes have now peaked around 70%. In part, this is due to a lack of useful biologic markers indicating which patients are likely to be cured and which will progress or relapse after standard treatment regimens. Once patients progress or relapse, very few will be cured by second-line treatment regimens. In this proposal, we aim to validate a new assay that detects tumor DNA from a simple peripheral blood draw as a surrogate for tumor burden in patients with Ewing sarcoma. We believe that this assay could help us to identify patients who are responding poorly to treatment very early in therapy and allow for patient-specific treatment modifications that could improve outcome for patients with refractory disease. Such an approach has been used to improve outcome for patients with pediatric leukemia where serial bone marrow samples are collected during therapy to determine patient response. For those patients with refractory leukemia, treatment intensification has significantly improved outcome. A similar approach for pediatric Ewing sarcoma has not been possible as repeated surgical biopsies are not feasible. In this proposal, we plan to validate the measurement of circulating tumor DNA assays obtained from blood samples as a biomarker of poor outcome for patients with Ewing sarcoma to usher in a new era of risk-stratified therapy for this disease.