Potential Treatment Identified for Inflammatory Myofibroblastic Tumors

The cause of a rare sarcoma may provide a promising new treatment target for soft tissue cancer.

Researchers may have pinpointed the cause of a rare soft tissue cancer that is resistant to chemotherapy and radiation. Furthermore, the team identified a drug that shows promise treating the disease.

Inflammatory myofibroblastic tumors are commonly found in children and young adults. The main form of treatment is surgery, however, the tumors will often reappear in different areas of the body. There is currently no standard of care for patients with this rare form of sarcoma.

In a study published in the Annals of Oncology, researchers found that the drug ceritinib showed promise in patients with inflammatory myofibroblastic tumors. Additionally, the researchers were able to trace tumor growth to chromoplexy.

Chromoplexy is a complex chromosomal rearrangement that causes genes to scramble, break DNA strands, and then reassemble in a defective way, according to the study.

A connection was made when a 32-year-old male patient did not respond to a nonsteroidal anti-inflammatory drug designed to shrink tumors in his chest, lung, and buttock. The FDA granted compassionate use for ceritinib, and within 2 weeks the patient began responding to the drug. After 18 months, the patient was well enough to undergo surgery to remove the tumors in his lung and buttock.

The researchers conducted a DNA test on the tumors using mate pair sequencing, a test that scans the entire genome to pinpoint genetic defects and chromosomal breaks. Using this method, researchers found 142 genes that had been impacted, many of which had known links to cancerous tumors.

“Mate pair sequencing helped identify these rearrangements, which may not have been seen with normal sequencing techniques,” said first study author Aaron Mansfield. “We look forward to offering more patients this new, advanced type of DNA testing to discover potential causes and treatments for diseases.”

The authors noted that more research on ceritinib needs to be done to determine whether it should be approved for individualized treatments.