Investigational Drug Effective in Treating Resistant GIST, Colorectal Cancer

Patients with GIST, a rare gastrointestinal sarcoma, benefited from major advancements in chronic myelogenous leukemia when Gleevec (imatinib) revolutionized therapy for both cancers a decade ago. Now, an experimental drug in metastatic colorectal cancer may have the same effect. Regorafenib, which targets two cancer cell pathways, extended survival in both cancers in two separate studies.

In the GIST study, progression-free survival was extended from less than one month on placebo to 4.8 months on regorafenib. Patients in the placebo group whose cancers progressed were allowed to receive regorafenib. Because patients were allowed to cross over from the placebo group to regorafenib, the trial did not produce a statistically significant overall survival difference; however, researchers noted a trend for longer survival with regorafenib.

In the CORRECT trial, patients with colorectal cancer who received regorafenib also saw an improvement in median overall survival (6.4 months versus 5 months).