Doctors in London say they have cured two babies of leukemia in the world’s first attempt to treat cancer with genetically engineered immune cells from a donor.
Experiments, which took place at London’s Great Ormond Street Hospital, raise the possibility of off-the-shelf cellular therapy using inexpensive supplies of universal cells that could be dripped into patients' veins on a moment’s notice.
The ready-made approach could pose a challenge to companies including Juno Therapeutics and Novartis, each of which has spent tens of millions of dollars pioneering treatments that require collecting a patient’s own blood cells, engineering them, and then re-infusing them.
Both methods rely on engineering T cells—the hungry predator cells of the immune system—so they attack leukemic cells.
The British infants, ages 11 and 16 months, each had leukemia and had undergone previous treatments that failed, according to a description of their cases published Wednesday in Science Translational Medicine. Waseem Qasim, a physician and gene-therapy expert who led the tests, reported that both children remain in remission.
Although the cases drew wide media attention in Britain, some researchers said that because the London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis. “It would be great if it works, but that just hasn’t been shown yet.”
Rights to the London treatment were sold to the biotech company Cellectis, and the treatment is now being further developed by the drug companies Servier and Pfizer.
Treatments using engineered T-cells, commonly known as CAR-T, are new and not yet sold commercially. But they have shown stunning success against blood cancers. In studies so far by Novartis and Juno, about half of patients are permanently cured after receiving altered versions of their own blood cells.
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Reposted via Next Big Future
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