November 23, 2017 |
Bladder Cancer Patient with Rare Genetic Mutations Shows Exceptional Response to Everolimus
March 13, 2014  | 

Boston, MA - A patient with advanced bladder cancer in a phase I trial had a complete response for 14 months to a combination of the targeted drugs everolimus and pazopanib, report scientists led by a Dana-Farber Cancer Institute researcher, and genomic profiling of his tumor revealed two alterations that may have led to this exceptional response.

This information can help identify cancer patients who may respond to everolimus, according to the report published in Cancer Discovery, a journal of the American Association for Cancer Research.

“Studying exceptional responders can help us understand the specific reasons why some tumors are highly sensitive to certain anticancer agents,” said Nikhil Wagle, M.D., of Dana-Farber Cancer Institute, the report’s first author. “We can use that information to identify patients whose tumors have genetic alterations similar to those found in exceptional responders, and treat them with those same agents.”

“We conducted a phase I clinical trial to test the efficacy of two anticancer agents — the mTOR inhibitor everolimus, and pazopanib, another drug that are approved for treatment of kidney cancers and sarcomas — and one of our patients developed near complete remission of his bladder cancer which lasted for 14 months,” said Wagle, who is also an Associate Member of the Broad Institute of MIT and Harvard. A complete response to a drug is when all signs of a tumor disappear.

“We performed whole-exome sequencing of the patient’s tumor, and to our surprise, we identified two mutations in the gene mTOR, which is the target for everolimus,” said Wagle. The protein made by this gene plays a role in many cell functions, and has been found to be mutated in a number of cancers. MTOR inhibitors such everolimus have been approved for treatment of some cancers, including breast and kidney. Continue>

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