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Molecule Disrupts DNA Sequence in Ewing's Sarcoma
Date:4/14/2008

Finding could yield first targeted therapy for the rare disease, researchers say

MONDAY, April 14 (HealthDay News) -- A molecule that disrupts the oncogene that causes Ewing's sarcoma has been identified by Georgetown University Medical Center researchers.

Ewing's sarcoma is a rare cancer that strikes children and young adults and can cause tumors to grow anywhere in the body. About 500 cases are diagnosed each year in the United States. Treatment involves a combination of five different chemotherapy drugs. An estimated 60 percent to 70 percent of patients survive over time, but many experience lingering effects from the chemotherapy.

Ewing's sarcoma occurs when there's an exchange of DNA between two chromosomes, a process called translocation. The EWS gene on chromosome 22 fuses to the FLI1 gene on chromosome 11, creating a new gene called EWS-FLI1, which produces the fusion protein that's causes cancer.

If further research shows that this newly identified molecule is effective, it could offer the first targeted therapy to treat Ewing's sarcoma, the researchers said. The molecule works through a so-called protein-protein interaction and could provide a model upon which to design other therapies.

"I think this holds really wonderful promise as a unique way of targeting fusion proteins," lead investigator Dr. Jeffrey Toretsky, a pediatric oncology physician and researcher at Georgetown University's Lombardi Comprehensive Cancer Center, said in a prepared statement.

"People thought it wasn't possible to have a small molecule that can bind between flexible proteins, but we have shown that it can be done," Toretsky said.

He noted that the study was conducted in laboratory cells, so further research is needed before the molecule can be tested in humans.

The study was to be presented Sunday at the annual meeting of the American Association for Cancer Research, in San Diego.


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