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UVa Oncologists Try New Approach to Target Childhood Cancer

October 3, 2013

Every day, 250 kids around the world die from cancer, and the fight to keep them alive is ongoing in Charlottesville.

Doctors at the University of Virginia Children's Hospital are using a growing trend to fight the cancer, and 8-year-old Norah Mastrandea is one patient who could potentially benefit.

"I don't think, until it touches your life, you really realize what childhood cancer is like and how it changes life for the child and the whole family," said Colleen Mastrandea, Norah's mother.

Norah is fighting a brain tumor called medulloblastoma. After a surgery to remove the tumor last year, another tumor grew while she was receiving chemotherapy. The cancer is still growing despite her strict regimen.

"For some reason, there's something about it that is not responding," Mastrandea said. "There's some pathway that we just haven't gotten quite right."

But doctors at UVa. Children's Hospital are trying a newer way to fight cancer called genomics. It allows doctors to see which specific genes are mutating and causing the cancer to grow and spread. They can then give their patients medicine that specifically targets the bad cells while leaving the good cells alone.

"We can target the cancer cell itself specifically and give better treatment without the side effects," said Dr. Colleen Druzgal, a pediatric oncologist and hematologist at the hospital.

Druzgal is Norah's oncologist. She says the hospital works with a company called Foundation Medicine that specializes in genomics. The company analyzes a patient's genes to target chemotherapies more effectively.

"There are some instances where if you can block that pathway, you can basically shut off the driving force that's driving the cancer," Druzgal said.

Norah recently took part in a clinical trial of a targeted agent. The third-grader is soft-spoken, worn out by the drugs that are fighting her cancer. But otherwise, she's a normal kid.

"She has a really funny sense of humor, and she knows how to, for somebody who's so young, really stick it to you," Druzgal said.

Norah described her own demeanor as tired and frustrated, but still happy.

"Maybe I'm going to get out of this place soon," she said.

For Norah's biological family and her adopted family at the hospital, the genomics offer some new hope thanks to science.

"We're coming at it from a different side," Druzgal said. "We're thinking about the biology of the tumor and why the medicines should work without having many many numbers to say that it will."

There's still some uncertainty, though, about how effective the drugs targeting the specific genes may be. The results can vary based on the type of cancer and the patient.

"We'll just kind of have to watch over time," Druzgal said. "It will take a while for us to know whether this is going to do anything. It's not going to be an overnight change."

But for the Mastrandeas, Norah's fight is part of a larger one against childhood cancer.

"I just really really look forward to seeing much, much more research on the genomics and figuring out the best and easiest way to treat these sweet angels," Mastrandea said. "They can't advocate for themselves, so we need to advocate for them."

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