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New Oral ‘Smart Drug’ Targets New Mutation And Dramatically Shrinks Aggressive Sarcoma And Lung Cancer

Posted Oct 28 2010 7:14pm

A new oral drug caused dramatic shrinkage of a patient’s rare, aggressive form of soft-tissue cancer that was driven by an abnormally activated protein, physician-scientists from Dana-Farber Cancer Institute report in the Oct. 28 issue of the New England Journal of Medicine.

A second patient who had a similar tumor that was not fueled by the mutant protein, called ALK (named for the first disease in which it was found, anaplastic lymphoma kinase), failed to respond to the drug, said the researchers, confirming the inhibitor’s specificity for the abnormal protein. The findings also highlight the value of “personalized medicine” gene-testing strategies to predict the best drug treatment for an individual’s particular, genetically defined cancer.

The patient described in the NEJM Brief Report is a 44-year-old man diagnosed in 2007 with inflammatory myofibroblastic tumor (IMT), a type of sarcoma that typically develops in the chest or abdomen in children and young adults.

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