Development and translation of novel therapies for pediatric sarcoma

Division Of Basic Sciences - Nci

Investigators

Linked publications, trials & patents

Abstract

In FY2017, we have identified several potential new agents for the treatment of pediatric-type sarcomas. We have published our findings on PEN-866, a novel HSP90 inhibitor-SN38 drug conjugate, which shows remarkable activity in preclinical models of rhabdomyosarcoma, Ewing sarcoma and osteosarcoma. We have subsequently opened a clinical trial to test this agent in the phase 1 setting and have recently begin to enroll patients. Additionally, we have identified several agents/combinations of agents that are active in Ewing sarcoma and are in the process of further studying their mechanisms preclinically.