The clinical trial I’m presenting is in a disease called dedifferentiated liposarcoma which is a relatively rare soft tissue sarcoma, several thousand cases a year in the United States. We have known for many years that there is a specific oncogene called cyclin-dependent kinase 4, or CDK4, that drives the growth of almost all these tumours. We’ve been interested in studying CDK4 inhibitors in this disease for over 15 years.

What was the methodology and findings?

Prior studies have shown that treatment with a CDK4 inhibitor can improve progression free survival but they have been small phase II studies. The purpose of this study was to conduct a large randomised phase III multicentre placebo-controlled study.

108 patients were enrolled across nine sites around the United States over a three-year period and they were randomised to receive either abemaciclib, the CDK4 inhibitor, or placebo. Patients who were randomised to placebo, if their cancer got worse they had the opportunity to cross over and receive abemaciclib. The primary endpoint of the study was to determine the median progression free survival.

What impact could these findings have in the clinic?

The study was substantially positive, the median progression free survival was 9.7 months with abemaciclib versus 1.5 months with placebo and response rate and overall survival were also better so this suggests that abemaciclib could become a new standard of care for the treatment of patients with recurrent dedifferentiated liposarcoma.