Adolescents and young adults with Ewing’s sarcoma (ES), a highly aggressive bone cancer, have few treatment options and a poor prognosis for recurrence. A multi-center Phase I/II clinical trial co-led by Joseph Ludwig, M.D., investigated the ability of the novel small molecule TK216 to disrupt the oncogenic fusion protein EWS-FLI1 in previously treated, relapsed/recurrent ES. In the study, 85 ES patients between the ages of 11 and 77 received TK216 by a continuous intravenous infusion.
Researchers observed two complete responses, one partial response and 14 patients with stable disease, demonstrating anti-tumor efficacy. One patient achieved a complete response following five relapses of ES, and the patient remains ES free for more than 55 months after study initiation. These findings suggest a potential path forward to investigate prospective drug candidates that target the pathognomonic fusion protein in ES and other sarcoma subtypes.