What is the current role of targeted therapies in treating sarcoma?

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RobertMakiMDPhD (Physician - Oncology - Hematology/Oncology (Verified) ) - 07 / 10 / 2012

One of the most common sarcomas, GIST, responds well to “kinase-targeted therapy”, a new type of drug that does not damage DNA but blocks protein function to try to make the cell commit suicide. These drugs have shown particular use recently in forms of leukemia and dramatically, but briefly, in melanoma. Other very rare sarcoma subtypes can respond to such kinase-directed agents, but the most common ones in adults do not respond so well to these drugs. That said, the drug pazopanib was just approved in the US and Europe for use in sarcomas except liposarcoma and GIST, since in these relatively unselected tumors there appeared to be at least a radiological benefit (tumor stabilization) with the use of pazopanib vs placebo. However, we do not know how pazopanib works in a given tumor, so it is a bit of an untargeted approach with a targeted agent. The term targeted therapy is misleading, as many of the drugs developed in the are indeed targeted as well, for example paclitaxel or vinorelbine targeting the microtubule or methotrexate poisoning a particular step in DNA synthesis. The targeting is also relative. We talk about “cleaner” or “dirtier” drugs based on how many targets a drug hits. More targets means more chances to do something effective, but greater chances for side effects too.
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