A Flip of a Genetic Switch: Stopping a Rare Childhood Cancer in Its Tracks

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A Flip of a Genetic Switch: Stopping a Rare Childhood Cancer in Its Tracks
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Researchers at Cold Spring Harbor Laboratory (CSHL) have identified a new drug target for Ewing sarcoma, a rare form of cancer typically diagnosed in children and young adults. Their experiments reveal that the cells responsible for this cancer can be effectively reprogrammed with the flick of a gen

Scientists discovered targeting a protein called ETV6 can turn Ewing sarcoma cancer cells back to normal-looking and -behaving connective tissue cells, as seen above. Credit: Vakoc lab/Cold Spring Harbor Laboratoryhave identified a new drug target for Ewing sarcoma, a rare form of cancer typically diagnosed in children and young adults. Their experiments reveal that the cells responsible for this cancer can be effectively reprogrammed with the flick of a genetic switch.

Ewing sarcoma causes tumors to grow in bones or the soft tissues surrounding them. Once a tumor begins to spread to other parts of the body, it can be very difficult to halt the disease’s progression. Even for patients with positive outcomes, treating Ewing sarcoma often causes toxic side effects. New treatments are badly needed, says CSHL Professor Christopher Vakoc, who led the research on ETV6.

Vakoc and his colleagues became excited about ETV6 when their experiments revealed that Ewing sarcoma cells seem uniquely dependent on this protein. “This protein is present in all cells. But when you perturb the protein, most normal cells don’t care,” he says. “The process by which the sarcoma forms turns this ETV6 molecule—this relatively innocuous, harmless protein that isn’t doing very much—into something that’s now controlling a life-death decision of the tumor cell.

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