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7-year-old girl beats leukemia - with disabled form of HIV

By Catholic Online (NEWS CONSORTIUM)
December 12th, 2012
Catholic Online (www.catholic.org)

Little Emily Whitehead's family had almost given up hope. Fighting leukemia for two years, Emily, or as she is commonly known, Emma relapsed for a second time during intensive chemotherapy treatment last February. Doctors had exhausted all the traditional treatments and her parents began to look at more radical options. Emily's salvation lay in a disabled form of HIV, the virus that causes AIDS.

LOS ANGELES, CA (Catholic Online) - The doctors at the Cancer Center at The Children's Hospital of Philadelphia suggested that Emily try a clinical trial that would use a disabled form of HIV to carry cancer-fighting genes into her T-cells. Doctors hoped that this would reprogram her immune system to recognize the cancer cells and start killing them.

Adult patients had responded well to the therapy in clinical trials at the Hospital of the University of Pennsylvania. The treatment, however was so new, her parents were warned that there would be attendant risks.

"We were told that we were down to 48 hours of making a decision or she could start having organ failure," her mother Mrs. Whitehead said. They soldiered on with the knowledge that even if the procedure didn't work, the information could help doctors save other sick children.

Emma became the first child to have the therapy known as CTL019 last April.

Emily became critically ill and was admitted to intensive care at the children's hospital. On April 24, doctors told her parents she had a one in 1,000 chance of surviving the night.

Then, trial leader Dr. Stephan Grupp and his team realized that the level of a certain protein had become very elevated as a result of the T-cells growing in Emily's body. The team administered the drug to Emily, with dramatic results. Emma's breathing improved, her fever dropped and her blood pressure was back to normal.

Emma's father was extremely proud of her daughter. "She told us from the beginning that she would continue to fight and do what we asked as long as we were there with her. We've stuck together as a team. She's definitely our hero."

In the experimental treatment, her T-cells were collected from her blood, then re-engineered in a lab to recognize and attach to a protein called CD19 that is found only on the surface of B cells. In order to do this, doctors used a gutted HIV virus, called a lentivirus, to carry special receptors into the T-cells. There is no risk of HIV infection from a lentivirus.

"Emily completely responded to her T-cell therapy. We checked her bone marrow for the possibility of disease again at three months and six months out from her treatment, and she still has no disease whatsoever," doctors said. "The cancer-fighting T-cells are still there in her body."

Emily went home in June and now enjoys going to school, playing football and walking her dog Lucy.

Scientists said that while the results were very promising, much more research needs to be done to see whether the therapy is a viable, safe and long-term solution for controlling certain cancers in children and adults.

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