Rhabdomyosarcoma, or rhabdo for short, is a childhood cancer that often turns deadly. It affects only a couple hundred kids in the United States each year, but a diagnosis can be devastating. Now, doctors are close to a treatment, and they have fruit flies to thank for it.
Sydney Mayrell loves crafts and cupcakes, but what she loves most is playing with her mommy.
Sydney and her mother have grown even closer since Sydney’s diagnosis of cancer three years ago.
"I felt a lump in her left thigh," said Erin Mayrell, Sydney’s mother.
That lump was rhabdo, an aggressive cancer that spreads through tissues in the body. For Sydney, it meant 54 weeks of chemotherapy, four weeks of radiation, and surgery.
"It can be a devastating disease. You’re faced with no choice but giving them the most aggressive kind of therapy you can give," said Dr. Rene L. Galindo, assistant professor of pathology, molecular biology and pediatrics at UT Southwestern Medical Center in Dallas.
Galindo hopes his research in fruit flies will change that. In the lab, he was able to show that silencing a specific gene in the flies prevented healthy cells from becoming cancerous and turned cancerous rhabdo cells back to normal. He was able to replicate the same results in human tumor cells.
"The cancer would stop being a tumor and it would become normal skeletal muscle," Galindo said.
The next step is a clinical trial in humans. If it works, it could essentially be a cure. That has Sydney and her mom excited. She’s in remission now, but this good news is icing on the cake.
Galindo said this method of gene silencing would offer a much less toxic and less harsh treatment for children. Rhabdo tumors usually occur in children under 6-years-old.