New treatments possible for a rare childhood cancer | NIH MedlinePlus Magazine

Thanks to new research, children with the rare childhood cancer rhabdomyosarcoma, or RMS, may be able to receive more targeted treatment.

Childhood RMS is a cancer of the soft tissue, like muscles and tendons. It accounts for about 3% of all pediatric cancers. Most cases of RMS are diagnosed in children and teens, with more than half in children under 10.

Scientists from the National Cancer Institute (NCI) and the Institute of Cancer Research in London looked at the DNA of tumors from 641 children with RMS. They were enrolled in several clinical trials over 20 years.

The scientists searched for genetic mutations that could help predict how well children with RMS would respond to treatment. They found mutations in several genes that seem to be connected to a more aggressive form of the disease and a lower chance of survival.

“These discoveries change what we do with these patients. They trigger a lot of really important research into developing new therapies that target these mutations,” said Javed Khan, M.D., of NCI’s Genetics Branch, who led the study.

The standard therapy for RMS involves a year of chemotherapy, radiation therapy, and surgery. That means children with this cancer get a lot of toxic treatments, said study co-author Jack Shern, M.D., of NCI’s Pediatric Oncology Branch.

“If we can predict who’s going to do well and who’s not, then we can really start to tailor our therapies or eliminate therapies that aren’t going to be effective in a particular patient,” Dr. Shern said. “For the children who aren’t going to do well, this allows us to think about new ways to treat them.”

Routine tumor genetic testing is a standard part of the treatment plan for more common cancers, such as breast cancer. The researchers hope that will soon be true for rare cancers such as RMS.

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