An experimental therapy has brought prolonged remissions to a high proportion of patients who were facing death from advanced leukemia after standard treatments had failed, researchers are reporting.
The therapy involves genetically programming cells from the patient’s own immune system to fight the disease.
The research included 30 patients: five adults ages 26 to 60, and 25 children and young adults ages 5 to 22. All were severely ill, with acute lymphoblastic leukemia, and had relapsed several times or had never responded to typical therapies. In more than half, the disease had come back even after a stem-cell transplant, which usually gives patients the best hope of surviving. Their life expectancy was a few months, or in some cases just weeks.
Six months after being treated, 23 of the 30 patients were still alive, and 19 of them have remained in complete remission.
The study, by researchers at the Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania, is being published in The New England Journal of Medicine.
“We have a number of patients who are a year or more out and are in remission and not requiring other therapies,” said Dr. Stephan A. Grupp, who led the part of the study done at Children’s Hospital of Philadelphia. He said those long remissions gave the researchers hope that the treatment would have lasting effects.
Earlier reports by the same researchers involved only a handful of patients, some with chronic rather than acute leukemia. The scientists say the growing number of patients treated helps demonstrate that the findings are real.
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