Researchers at Mayo Clinic Center for Individualized Medicine have discovered a potential cause and a promising new treatment for inflammatory myofibroblastic tumors, a rare soft tissue cancer that does not respond to radiation or chemotherapy.
New research from Aaron Mansfield, MD, an oncologist at Mayo Clinic, and George Vasmatzis, PhD, the Co-Director of the Biomarker Discovery Program of Mayo Clinic Center for Individualized Medicine, finds the drug ceritinib shows promise as a new treatment for inflammatory myofibroblastic tumors (Ann Oncol 2016;27(11):2111-17). The study also traced tumor growth to chromoplexy: a complex chromosomal rearrangement that causes genes to scramble, break DNA strands, and then reassemble in a defective way.
Investigators made the connection when a 32-year-old man failed to respond to a nonsteroidal anti-inflammatory drug to shrink tumors in his lung, chest, and buttock. Because there were no available clinical trials, researchers sought and gained FDA approval for compassionate use of ceritinib. Within 2 weeks, the patient started responding to the drug. After 18 months, he was well enough to undergo surgery to remove tumors from his lung and buttock. Mayo researchers were able to conduct a new DNA test on the tumors known as mate pair sequencing. They found 142 genes had been impacted, many of which have known links to cancerous tumors.
"Mate pair sequencing helped identify these rearrangements, which may not have been seen with normal sequencing techniques," noted Mansfield. "We look forward to offering more patients this new, advanced type of DNA testing to discover potential causes and treatments for diseases."
The main form of treatment for patients with an inflammatory myofibroblastic tumor is surgery, but the tumors often reappear in different parts of the body. There is no standard of care for patients with inflammatory myofibroblastic tumors, thus making the discovery of tumor response to ceritinib all the more important. Mayo Clinic investigators are recommending further study of this drug to determine whether it should be approved for individualized treatments.