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Gene Therapy Transforms Immune System Into Cancer Killer in U.S. Study

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Gene therapyU.S. government researchers are expanding use of gene therapy to fight cancer, turning the human immune system against a deadly tumor found in young adults.

The approach may shrink tumors in many patients with common cancers, said Steven Rosenberg, chief of surgery at the National Cancer Institute’s Center for Cancer Research in Bethesda, Maryland. In a study, 9 of 17 patients with advanced cancer that had withstood other treatments saw tumors shrink after gene therapy, according to results published today in the Journal of Clinical Oncology.

The researchers genetically modified each patient’s immune system so it would recognize antigen produced by cancer cells and destroy them. The approach worked five years ago in a small study that targeted melanoma. The new work uses an antigen produced by many tumors, including some in breast, prostate, lung and ovarian cancer, though only certain patients with synovial cell sarcoma and metastatic melanoma were included in the study.

“It’s not a vial off the shelf,” Rosenberg, the lead investigator for the study, said in a telephone interview. “It’s the ultimate in personalized medicine because we are making a new drug for every patient.”

The treatment takes a patient’s white blood cells, called lymphocytes, amplifies their number and activity, and gives them back, Rosenberg said.

“We have now shown that this gene therapy, this genetic engineering of the immune system, can work not only on melanoma but on sarcoma,” he said. “We’re now looking to see if it will work on other cancers.”

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