Breakthrough T cells treatment effective against leukaemia

August 11, 2011 04:26 pm | Updated August 12, 2011 01:59 pm IST - Washington

This microscopy image shows immune system T-cells, center. Scientists are reporting the first clear success with gene therapy to treat leukemia, using the patients' own blood cells to hunt down and wipe out their cancer. Photo: AP

This microscopy image shows immune system T-cells, center. Scientists are reporting the first clear success with gene therapy to treat leukemia, using the patients' own blood cells to hunt down and wipe out their cancer. Photo: AP

In what is being hailed as a cancer treatment breakthrough, three men suffering late-stage leukemia were cured in just three weeks after being treated with genetically engineered versions of their own immune cells.

The findings are the first demonstration of the use of gene transfer therapy to create T cells aimed at cancerous tumours.

“Within three weeks, the tumours had been blown away, in a way that was much more violent than we ever expected,” said senior author Carl June, MD, director of Translational Research and a professor of Pathology and Laboratory Medicine in the Abramson Cancer Center, who led the work. “It worked much better than we thought it would.” Researchers believe that the breakthrough could provide a ‘roadmap’ for the treatment for cancers of the lungs and ovaries, myeloma, which attacks the bone marrow, and melanoma, the most aggressive form of skin cancer The results of the pilot trial of three patients are a stark contrast to existing therapies for CLL. The patients involved in the new study had few other treatment options. The only potential curative therapy would have involved a bone marrow transplant, a procedure which requires a lengthy hospitalization and carries at least a 20 percent mortality risk - and even then offers only about a 50 percent chance of a cure, at best.

“Most of what I do is treat patients with no other options, with a very, very risky therapy with the intent to cure them,” says co-principal investigator David Porter, MD, professor of Medicine and director of Blood and Marrow Transplantation. “This approach has the potential to do the same thing, but in a safer manner,” he added.

Gene transfer therapy using T cells has been a holy grail of treatment for various cancers over the past 25 years but has proved very difficult to achieve in human trials.

The researchers say their success in activating the T cells, making them multiply and then kill the cancer cells in this type of cancer, is “unprecedented”.

The study was recently published in the New England Journal of Medicine and Science Translational Medicine.

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