Margaret von Mehren, MD
GIST Research Laboratory
The primary goal of our work is the testing and evaluation of new therapies for sarcomas. There are approximately 7,000 new cases of sarcomas diagnosed yearly with only 50% surviving their disease. Current therapies do not lead to cures when sarcomas return. Our group has focused on testing new therapies in advanced sarcomas. Trials testing targeted therapies in the appropriate biologic context is sought. Examples of such trials are the sarc-kinase inhibitor dasatanib and IGF-1R for sarcomas.Top
We have a particular interest in novel therapies for Gastrotintestinal Stromal Tumors (GIST). GIST are characterized by the presence of activating mutations, most commonly in KIT and PDGFRA. We participated in the initial trials of imatinib and sunitinib. We have been involved in testing many other kinase inhibitors as well as novel therapeutic agents such as heat shock protein inhibitors. Our interest in GIST is supplemented by pre-clinical studies conducted in the GIST Research Laboratory. Studies in the laboratory identified IGF-1R as a potential target in GIST lacking an activating mutation. In vitro studies suggest activity of IGF-1R inhibition alone and in combination with KIT inhibition has anti-proliferative and pro-apoptotic effects. We will be testing the benefit of IGF-1R inhibitors in GIST.