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New Gene Therapy is Saving Lives

A seven year old girl from Philipsburg, Pennsylvania was on the verge of death from leukemia when doctors at the University of Pennsylvania gave her an injection of her own genetically modified cells and were able to save her life.

Emily Whitehead, 7

According to The Philadelphia Inquirer, the lifesaving drama began for young Emily Whitehead when her cancer-wracked body stopped responding to all available treatments.Diagnosed with leukemia at the age of five, she should have had an 85 percent chance of beating the cancer, but nothing ever seemed to work as usual for the pretty little brown-eyed girl. Massive batteries of chemotherapy and a bone-marrow transplant never brought any more than brief periods of remission.

By March, 2012, "she was out of options," her father told the Inquirer.

Doctors decided to try a promising new therapy that involves genetically engineering a patient's own immune system cells known as T-cells. These cells are "reprogrammed" to make them attack B-cells, which is the blood component that turns malignant in both chronic lymphocytic leukemia and acute lymphoblastic leukemia.

Emily was given three intravenous T-cell doses and, once again, things didn't progress normally. Instead, Emily's blood pressure dropped dangerously low, her fever spiked to 105 and she began suffering hallucinations and difficulty breathing.

Doctors tried to intervene with a ventilator and medications such as morphine and steroids, but nothing worked.

"Emily may not make it through the night," wrote her mother, Kari, on the fifth day of Emily's ordeal.

Teetering on the verge of death, a new lab analysis revealed that Emily's blood contained high levels of a chemical known as interleukin 6. Doctors frantically began searching the medical literature or any drug designed to block it. They found a new rheumatoid arthritis drug known as Actemra, and quickly gave it to Emily.

Within hours, her vital signs were stabilized. Three weeks after the therapy began, she was completely free of the cancer that had been on the verge of killing her.

Not only was her life saved, but the lives of many others as well, thanks to the lessons learned after Emily's experience.

The new gene therapy has been tried on 10 patients thus far, seven of which went into remission, but several of these patients also got sick after receiving the T-cells.

"Previous patients got sick, but it wasn't clear at the time whether it was due to the T cells or an infection," said pediatric oncologist Stephan Grupp, who oversaw Emily's care at Children's Hospital of Philadelphia. "Now we know the main reason they were sick was the cells. But now we can intervene. She taught us."

Without the discovery of how to treat the high interleuken 6 levels in the blood of patients who receive the T-cells, the therapy might have eventually been discarded as too risky. However, now doctors know how to intervene if the side-effects of the drug prove as catastrophic as they did in Emily's case.

The Penn research team, which is being led by gene-therapy pioneer Carl June, presented results from the first 10 patients at a medical conference this past weekend.

"Of the 10, only two patients have not gotten better on the T cell therapy, even though all had stopped responding to conventional treatments," the Inquirer reports. "Four patients have had a complete response - their leukemia was eradicated - with the longest so far lasting 28 months. In four other patients, leukemia diminished dramatically."

As for Emily, she's grateful for the new lease on life. The last time she was at the hospital, she wrote on a whiteboard: "Thanks for the T cells," followed by the names of her doctors.

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