PHILIPSBURG, Pa. Emma Whitehead has been bounding around the house lately, practicing somersaults and rugby-style tumbles that make her parents wince.
It is hard to believe, but last spring Emma, then 6, was near death from leukemia. She had relapsed twice after chemotherapy, and doctors had run out of options.
Desperate to save her, her parents sought an experimental treatment at the Childrens Hospital of Philadelphia, one that had never before been tried in a child or in anyone with the type of leukemia Emma had. The experiment, in April, used a disabled form of the AIDS virus to reprogram Emmas immune system genetically to kill cancer cells.
The treatment nearly killed her. But she emerged from it cancer-free and seven months later is still in complete remission. She is the first child and one of the first humans ever in whom new techniques have achieved a long-sought goal giving a patients own immune system the lasting ability to fight cancer.
She is among just a dozen patients with advanced leukemia to have received the experimental treatment, which was developed at the University of Pennsylvania. Similar approaches are being tried at other centers.
Our goal is to have a cure, but we cant say that word, said Dr. Carl June, who leads the research team at the University of Pennsylvania. He hopes the new treatment will eventually replace bone-marrow transplantation.
Three adults with chronic leukemia treated at the University of Pennsylvania have also had complete remissions, with no signs of disease; two of them have been well for more than two years, said Dr. David Porter.
Four adults improved but did not have full remissions, and one was treated too recently to evaluate. A child improved and then relapsed. In two adults, the treatment did not work at all.
The Pennsylvania researchers are presenting their results Sunday and Monday in Atlanta at a meeting of the American Society of Hematology.
Despite the mixed results, cancer experts not involved with the research say it has tremendous promise.
This is a major breakthrough, said Dr. Ivan Borrello, a cancer expert and associate professor of medicine at the Johns Hopkins University School of Medicine.
A major drug company, Novartis, is betting on the Penn team and has committed $20 million to building a research center on the Penn campus to bring the treatment to market.
Herve Hoppenot, president of Novartis Oncology, called the research fantastic and said it had the potential if the early results hold up to revolutionize the treatment of leukemia and related blood cancers.
To perform the treatment, doctors remove millions of the patients T-cells a type of white blood cell and insert new genes that enable the T-cells to kill cancer cells. The new genes program the T-cells to attack B-cells, a normal part of the immune system that turns malignant in leukemia.
The altered T-cells called chimeric antigen receptor cells are then dripped back into the patients veins, and if all goes well they multiply and start destroying the cancer.
The T-cells home in on a protein called CD-19 that is found on the surface of most B-cells, whether they are healthy or malignant.
A sign that the treatment is working is that the patient becomes terribly ill, with raging fevers and chills a reaction that oncologists call shake and bake, June said. Its medical name is cytokine-release syndrome.
The storm can also flood the lungs and cause perilous drops in blood pressure effects that nearly killed Emma.
Steroids sometimes ease the reaction but did not help Emma. Her temperature hit 105. She wound up on a ventilator, unconscious and swollen almost beyond recognition, surrounded by friends and relatives who had come to say goodbye.
But at the eleventh hour, a battery of blood tests gave the researchers a clue as to what might help save Emma: Her level of one of the cytokines, interleukin-6 or IL-6, had shot up a thousandfold.
Doctors had never seen such a spike before and thought it might be what was making her so sick. June knew that a drug could lower IL-6 his daughter takes it, for rheumatoid arthritis. Emmas oncologist, Dr. Stephan Grupp, ordered the drug. The response, he said, was amazing.
Within hours, Emma began to stabilize. She woke up a week later, on May 2, the day she turned 7.
Since then, the research team has used the same drug, tocilizumab, in several other patients.
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