GIST Support Wiki

Regorafenib for Colon Cancer and GIST

January 19, 2012


Here are the results of the phase III regorafenib trial for colon cancer. Evidently these results are good enough to warrant an application to the FDA for market approval,. I'm keeping my fingers crossed that the results for regorafenib and GIST are much stronger than the data for colon cancer.

""The investigational drug regorafenib extended overall survival in patients with treatment-refractory advanced colorectal cancer by 1.4 months in a phase III trial, according to its manufacturer.""

•Median progression-free survival was 1.9 months with regorafenib versus 1.7 months for placebo (P<0.000001) •Disease control rate was 44.8% with regorafenib versus 15.3% with placebo (P<0.000001) •Objective response rate was 1.0% with regorafenib versus 0.4% with placebo (P=0.188)


Regorafenib has been in phase III trial for GIST. Lee Ann is in the trial. Anecdotally, there have been favorable comments from the trial pts, so we can stay hopeful. Usually results of such important trials are released only at major meetings such as ASCO, so we can expect that to be the case for our trial.

Regorafenib extended overall survival in the colon cancer population by a median of six weeks compared to placebo. I think regorafenib, which is a KIT inhibitor, is a more direct match to the driving molecular defect of GIST than it is to colon cancer's--where I think the effects would be on angiogenesis primarily.