Rhabdomyosarcoma discoveries move from lab to clinic

St. Jude scientists have completed the most complete analysis yet of the muscle and soft tissue tumor rhabdomyosarcoma. The researchers found weaknesses they can target. They also discovered a promising precision medicine for the disease.

Cure rates are 75 percent for patients whose rhabdomyosarcoma has not spread. But for children whose cancer returns or spreads, long-term survival is 30 percent or less.

Researchers learned that the two main varieties of rhabdomyosarcoma occur at different developmental times.

They also found several signaling pathways that are disrupted in tumor cells. A drug called AZD1775 inhibits an enzyme in one of these pathways. Scientists screened more than 1,700 drug-tumor combinations as part of the project. As a result, the Children’s Oncology Group expanded a clinical trial of AZD1775 to include children with high-risk rhabdomyosarcoma.

The research, part of the St. Jude–Washington University Pediatric Cancer Genome Project, appeared in the journal Cancer Cell.

Read the news release.