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It is used in the treatment of chromic myelogenous leukemia CML, a relatively rare cancer. Before Gleevec, CML killed Americans and after Gleevec, in , it only killed people — all using an oral medication with virtually no side effects. This is a truly amazing drug, so dramatically successful that it was thought to be the herald of an entirely new era of chemotherapy. Gleevec was supposed to be the beginning, not the end. But like any one-hit wonder, the first turned out to be the best. Genetic treatments will work wonders for genetic diseases, but the specific circumstances surrounding CML did not apply to most of the common cancers breast, colorectal, prostate that we face today. CML is almost completely a genetic disease caused by the mixup of chromosomes during cell growth. Normally, when cells divide, they provide exactly the same chromosomes to each new cell. However, in CML a piece of chromosome 9 ended upon chromosome 12 and vice versa. Virtually all patients with CML had this Philadelphia chromosome, and in , it became clear that this genetic aberration caused the cancer. This protein is a signalling molecule called a kinase, which works like an accelerator on cell growth.
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