In a small trial of advanced chronic lymphocytic leukemia (CLL) patients, genetically modified versions of their own T cells behaved like "serial killers" and hunted down and obliterated tumors, resulting in sustained remissions of up to a year.
The breakthrough gene therapy treatment has been 20 years in the making, and provides a roadmap for other cancers, say researchers from the University of Pennsylvania's Abramson Cancer Center and Perelman School of Medicine, who report the results in a study published today, 11 August, in two journals: the New England Journal of Medicine and Science Translational Medicine.
The pilot trial of three patients is the first to show how gene transfer therapy can create "serial killer" T cells aimed at cancerous tumors, and the team, led by senior author Dr Carl June, director of Translational Research and a professor of Pathology and Laboratory Medicine in the Abramson Cancer Center, believe the protocol they have developed can be used to develop treatments for other cancers, including ovarian and lung cancers, and myeloma and melanoma.
The three patients in the trial had advanced CLL and their only hope of a cure was a bone marrow transplant, a procedure that requires long stays in hospital and carries a 1 in 5 chance of death. Even then, the chance of a cure is at best 50%, said the researchers.
For the study, the researchers removed patients' own T cells (a type of white blood cell), modified them in Penn's vaccine production labs, then infused them back into the patients' bodies after chemotherapy.
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