Twenty years of work to get to a gene therapy against leukemia and now the first, unexpected success. Have been described as "by far higher than expected" results obtained from a group of American scientists at the University of Pennsylvania in three patients at an advanced stage chronic lymphocytic leukemia. Severely ill patients, for which there was no other hope but a bone marrow transplant with a mortality risk greater than 20% and probability of success less than 50%.
But doctors have tried another way: the use of T lymphocytes taken from patients and genetically modified in order to get the "serial killer GMOs" armed against cancer cells. The answer came within three weeks of treatment. Two of the patients in remission for a year. And in the third seems to be no more evidence of disease. These results are still preliminary, the experiment must continue and expand, the authors speculate, but the pilot study allows to hope.
The data of the mini-trial, funded by the Alliance for gene therapy of cancer, are published on
New England Journal of Medicine and Science of Translational Medicine. The results demonstrate for the first time that the way of gene therapy against leukemia is feasible.
Researchers dell'Abramson Cancer Center and the Perelman University of Pennsylvania School of Medicine have used T cells taken from patients' immune systems.
Before being reinfused into "owners, these cells have been reprogrammed through a viral vector, to produce a protein receptor (Car) that engages a particular structure present on diseased cells (CD19 antigen). A classic joints like "lock and key", which has allowed the "GM Killer" to grab the leukemia cells and destroy them.
Now scientists hope to apply the same approach in children with leukemia in which conventional treatments have failed, and against other cancers including non-Hodgkin's lymphoma, acute lymphocytic leukemia, mesothelioma and cancers of the ovary and pancreas.