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Science Translational Medicine

  1. Researchers Announce Successful Clinical Trial Of Gene Therapy Treatment For Leukemia

    For the first time, researchers have successfully used gene therapy to treat a form of leukemia called chronic lymphoblastic leukemia. The clinical trial was only conducted in three patients, which is such a small sample size that it is far too soon to be declaring victory over cancer, but it is an encouraging breakthrough. The research is described in two papers, published in the New England Journal of Medicine and Science Translational Medicine. People have been talking about gene therapy for more than twenty years. Though it holds immense potential, researchers have run into problems with gene therapy as a treatment. In previous research, therapeutic genes that are inserted in a specific place tended to move around for reasons that researchers have struggled to understand. The goal of gene therapy is for a gene that is inserted into a specific place to stay in that spot to serve out its function in the cell. With the new leukemia treatment, this is exactly what the researchers were able to achieve.

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  2. Fighting Cancer With Specially Trained Cells

    Researchers looking to treat cancer patients without subjecting them to the ravages of chemotherapy have published some promising results in the journal of Science Translational Medicine on "adoptive T-cell therapy." Using this technique, researchers removed cells from nine melanoma patients immune system that fight disease, called T-cells. They then "trained" the cells, by exposing them to genetically engineered cells that carried tumor antigens, which signaled the T-cells to attack. The new, smarter, more experienced cancer-fighting cells were then multiplied and re-introduced to the patient's body. After two weeks, the cancer in four of the nine patients had stabilized, neither growing nor shrinking. In one patient, the cancer had disappeared entirely and was still cancer-free after two years. Five of the nine patients also responded much better to cancer drug treatments later on. Though this is only an early study, and will require many more experiments before it can be considered for widespread use, it does bode well for researchers. Especially, since previous T-cell training experiments failed when the cells died off when put back in the body. With any luck, future research will be just as promising as this study. (via Discover, image via Wikipedia)

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