How fruit flies are helping doctors figure out how to treat this little girl's cancer.
It's called Rhabdo for short, a childhood cancer that often turns deadly.
It only affects a couple hundred kids in the U.S. 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...
"...to play with mommy."
This mom and daughter have grown even closer since Sydney's diagnosis of cancer three years ago.
"I felt a lump in her left thigh," said Erin, Sydney’s mom.
That lump was Rhabdo, an aggressive cancer that spreads through tissues in the body. For Sydney it meant 54 weeks of chemo, four weeks of radiation, and surgery!
"It can be a devastating disease,” explains Doctor Rene Galindo, Assistant Professor of Pathology, molecular biology and pediatrics at UT Southwestern Medical Center. ”You're faced with no choice but giving them the most aggressive kind of therapy you can give."
But Doctor 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," said Galindo.
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!
Doctor Galindo says this method of gene silencing would offer a much less toxic and less harsh treatment for children.
Rhabdo tumors usually occur in children under six years old.
TOPIC: FRUIT FLIES FOR CANCER: MEDICINE'S NEXT BIG THING?
REPORT: MB # 3629
BACKGROUND: Rhabdomyosarcoma is a type of sarcoma, which is cancer of soft tissue, connective tissue, or bone. It is the most common type of soft tissue sarcoma in children and can begin in many places in the body. However, it usually begins in muscles that are attached to bones and that help the body move. Soft tissues of the body connect, support, and surround other organs and body parts. Soft tissues include: nerves, fat, blood vessels, lymph vessels, fat, muscles, tendons, and synovial tissues (tissues around joints). There are three main types of rhabdomyosarcoma: embryonal, alveolar, and anaplastic. Embryonal, which is the most common type, occurs most often in the head and neck area or in the urinary or genital organs. Alveolar occurs in the arms or legs, chest, genital organs, abdomen, or anal area, and it usually occurs in the teen years. Anaplastic is a rare type that occurs in children. (Source: www.cancer.gov)
RISK FACTORS: Risk factors include the following inherited diseases: Noonan syndrome, Costello syndrome, Beckwith-Wiedemann syndrome, pleuropulmonary blastoma, neurofibromatosis type 1, and Li-Fraumeni syndrome. Children who had a high birth weight or were bigger than expected at birth have an increased risk of embryonal rhabdomyosarcoma. (Source: www.cancer.gov)
SYMPTOMS: Childhood rhabdomyosarcoma can cause lumps and other symptoms, but the symptoms depend on where the cancer forms. Symptoms can include bulging of the eye, blood in the urine, bleeding in the nose, throat, vagina, or rectum, headache, trouble urinating or having bowel movements, and swelling that keeps getting bigger and more painful. (Source: www.cancer.gov)
NEW TECHNOLOGY: The traditional treatments of chemotherapy, surgery, and radiation for rhabdomyosarcoma can result in disfigurements and disabilities. The newest breakthrough in improving treatments comes from UT Southwestern Medical Center. Researchers identified the role for a gene, called TANC1, to convert normal muscle cells in fruit flies into cells that behave as aggressive Rhabdo-type cells. By silencing the gene, researchers convert healthy cells to a non-cancerous state. "This suggests a completely different way of caring for Rhabdo - by targeting this particular gene. If we take down TANC1 activity, it essentially rescues these cells from their neoplastic state and gets them to complete their developmental process," Dr. Rene Galindo was quoted as saying. In a previous fruit fly study, the Galindo lab learned that over-expression of a gene, called PAX-FOXO1, caused muscle cells to improperly fuse. The newer study revealed that PAX-FOXO1 is dependent on TANC1 in making muscle cells go awry. (Source: http://www.utsouthwestern.edu/newsroom/center-times/year-2012/may/galindo-research-rhabado.html)
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FOR MORE INFORMATION, PLEASE CONTACT:
Rene L. Galindo, MD, PhD
Assistant Professor of Pathology, Molecular Biology, and Pediatrics
UT Southwestern Medical Center
If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Andrew McIntosh at firstname.lastname@example.org.