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In what’s being billed as a medical first, scientists at the Great Ormond Street Hospital in London have presented a hopeful solution to cancer by developing a novel method of gene editing.

Cancer continues to be one of the major diseases that plagues humanity. Around the world, approximately 1 in 6 deaths is due to cancer, according to the WHO. The prevalence of cancer is due, in part, to the absence of a universal cure for all forms of the disease.
The team combined a novel but promising cell therapy called chimeric antigen receptor T cell (CAR T) with a gene-editing technique called TALENS to treat two infants with an aggressive form of leukemia.

In a study appearing in the journal Science Translational Medicine, the researchers reported their treatment has succeeded in keeping the two infants cancer-free for 16 and 18 months, respectively.

The technique combines CAR-T’s ability to use a patient’s immune system to fight cancer, and TALENS that allows for direct manipulation of the patient’s genes.

In a CAR T cell therapy, specialized immune cells (T cells) are taken from a patient’s blood and given special surface receptors called CARs. Once ready, the CAR T cells are infused back into a patient’s blood, where they can attach to tumors and destroy them. In

the study, the doctors made four genetic changes to T cells from donors, which they hoped would prove successful for treating their young patients’ cancers.

Clinical trials have now demonstrated the effectiveness of CAR T cell therapy, however, as every set of T cells must to be tailored to each individual patient, it’s proven to be a tedious and expensive process.

Gene-editing, however, would help make it easier to develop universal T cells.
The British infants, ages 11 and 16 months, each had leukemia and had undergone previous treatments that failed. 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. “It would be great if it works, but that just hasn’t been shown yet.”

Bottom line is that it will still take time and many more trials before this treatment combo could be made available to more patients, or even patients with cancers other than leukemia.

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