Advanced GIST is a rare but usually lethal cancer


GIST is a rare intestinal soft tissue tumor. About 10 new GISTs are diagnosed annually per 1 million in most populations. The majority of GISTs contain a mutation in KIT or platelet-derived growth factor alpha-receptors. The mutation activates receptors abnormally and enables the continuous growth and dividing of tumor cells. About 60% of patients are treated with surgery alone. The rest of the patients, who have metastatic disease or whose tumor has a high risk of recurrence are treated with KIT and platelet-derived growth factor receptor alpha targeting small molecule compounds such as Glivec®.  These therapeutics prevent oncogenic function of the mutated receptors and kill tumor cells. However, most GISTs become resistant to the current therapies eventually and novel approaches to treat cancer are needed.