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August 12, 2011

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Scientists hail new gene treatment for leukemia

SCIENTISTS are reporting the first clear success with a new approach for treating leukemia - turning the patients' own blood cells into assassins that hunt and kill cancer cells.

They have achieved this in only three patients so far, but the results are striking - two appear cancer-free up to a year after treatment, and the third is improved but still has some cancer. Scientists are preparing to try the same gene therapy on other kinds of cancer.

Dr Carl June, a gene therapy expert at the University of Pennsylvania, said: "We were surprised it worked as well as it did. We are just a year out now. We need to find out how long these remissions last."

June led the study, announced this week in two publications - New England Journal of Medicine and Science Translational Medicine. It involved three men with advanced cases of lymphocytic leukemia. Currenly, the only hope of a cure is bone marrow or stem cell transplants, which do not always work.

Scientists have been trying for years to find ways to boost the immune system's ability to fight cancer. Earlier attempts at genetically modifying bloodstream soldiers called T-cells had limited success - the cells did not reproduce well and quickly disappeared.

June and his colleagues adapted the technique, using a new carrier to deliver altered genes to the T-cells and a signaling mechanism telling the cells to multiply and kill.

That resulted in armies of "serial killers" targeting cancer cells, destroying them, and killing new cancer as it emerged. It is known that T-cells attack viruses this way, but it is the first time it has been achieved against cancer, June said.

Blood was taken from each patient and T-cells removed, altered in a laboratory, and returned to the patient by infusion The researchers described the experience of one 64-year-old patient in detail. There was no change for two weeks, then he became ill with chills, nausea and fever. He and the other two patients suffered a condition that occurs when a large number of cancer cells die at the same time - a sign the gene therapy is working.

"It was like the worse flu of their life," June said. "But after that, it is over. They are well."

The main complication seems to be that the technique also destroys some other infection-fighting blood cells, for which patients require monthly treatments.



 

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