Abstract
Ewing sarcoma is a small round blue cell malignancy arising from bone or soft tissue and most commonly affects adolescents and young adults. Metastatic and relapsed Ewing sarcoma have poor outcomes and recurrences remain common. Owing to the poor outcomes associated with advanced disease and the need for a clear research strategy, the Children’s Oncology Group Bone Tumor Committee formed the New Agents for Ewing Sarcoma Task Force to bring together experts in the field to evaluate and prioritize new agents for incorporation into clinical trials. This group’s mission was to evaluate scientific and clinical challenges in moving new agents forward and to recommend agents and trial designs to the Bone Tumor Committee. The task force generated a framework for vetting prospective agents that included critical evaluation of each drug by using both clinical and non-clinical parameters. Representative appraisal of agents of highest priority, including eribulin, dinutuximab, cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitors, anti-angiogenic tyrosine kinase inhibitors, and poly-ADP-ribose polymerase (PARP) inhibitors, is described. The task force continues to analyze new compounds by using the paradigm established.
Highlights
Ewing sarcoma (ES) is a small round blue cell bone tumor most commonly occurring in adolescent and young adult (AYA) patients
We summarize our work to date, including establishing a framework to prioritize potential agents in this rare disease, characterizing challenges in understanding the Clinical criteria From a clinical perspective, the paramount criterion is a signal of activity in early-phase testing with agents meeting this criterion prioritized for rapid translation into a trial for relapsed or newly diagnosed metastatic populations
Additional key clinical trial design considerations In addition to establishing recommendations for advanced ES cytotoxic backbones, we examined other important aspects of trial design including patient randomization, maintenance therapy, and specific differences in trial design approach for patients with upfront metastatic versus relapsed ES
Summary
Committee formed the New Agents for Ewing Sarcoma Task Force to bring together experts in the field to evaluate and prioritize new agents for incorporation into clinical trials. This group’s mission was to evaluate scientific and clinical challenges in moving new agents forward and to recommend agents and trial designs to the Bone Tumor Committee.
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