The first attempt in the United States to use a gene editing tool called CRISPR against cancer seems safe in three patients who've had it so far, but it's too soon to know if it will improve survival, doctors reported Wednesday. The doctors were able to take immune system cells from the patients' blood and alter them genetically to help them recognize and fight cancer, with minimal and manageable side effects, the AP reports. The treatment deletes three genes that might have been hindering these cells' ability to attack the disease, as well as adds a new, fourth feature to help them do the job. "It's the most complicated genetic, cellular engineering that's been attempted so far," said study leader Dr. Edward Stadtmauer of the University of Pennsylvania. Two of the patients have multiple myeloma, a blood cancer, and the third has a sarcoma, cancer that forms in connective or soft tissue.
All had failed multiple standard treatments. Their blood was filtered to remove immune system soldiers called T cells, which were modified in the lab and then returned to the patients through an IV. The cells should multiply into an army within the body and act as a living drug. So far, the cells have survived and have been multiplying as intended, Stadtmauer said. After two to three months, one patient's cancer continued to worsen and another was stable; the third patient was treated too recently to know how she'll fare. The plan is to treat 15 more patients and assess safety and how well it works. The early results were released by the American Society of Hematology; details will be given at its annual conference next month. Several study leaders and the university have a financial stake in the company and may benefit from patents and licenses on the technology.
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