Scientists for the first time have used gene therapy to successfully destroy cancer tumors in patients with advanced disease
Researchers at the University of Pennsylvania engineered patients' own pathogen-fighting T-cells to target a molecule found on the surface of leukemia cells.
The altered T-cells were grown outside of the body and infused back into patients suffering from late-stage chronic lymphocytic leukemia (CLL), which affects the blood and bone marrow and is the most common form of leukemia.
Two participants in the Phase I trial have been in remission for up to a year. A third had a strong anti-tumor response, and his cancer remains in check. The research group plans to treat four more patients with CLL before moving into a larger Phase II trial.
"We put a key onto the surface of the T-cells that fits into a lock that only the cancer cells have," said Dr. Michael Kalos, director of translational and correlative studies at the University of Pennsylvania's Perelman School of Medicine and an investigator on the study.
The results provide "a tumor-attack roadmap for the treatment of other cancers," including those of the lung and ovaries as well as myeloma and melanoma, researchers said.
The findings were published simultaneously Wednesday in the New England Journal of Medicine and Science Translational Medicine.
Kalos said past efforts to use the technique, known as "adoptive T-cell transfer," failed either because the T-cell response was too weak or proved too toxic for normal tissue.
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