Doctors Use Gene-Edited Immune Cells to Treat Leukemia Patients

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Two babies who were suffering from the blood cancer leukemia have been cured this month with thanks to a group of British doctors using a treatment involving the use of genetically modified immune cells. Although the babies have only been cured for 16 and 18 months, it’s still a huge breakthrough nonetheless.

What makes this study particularly exciting is that uses a combination of CAR T cell therapy and a new gene-editing technique called TALENS to free the patient from the disease. It’s not the first time CAR T’s been used as part of an immunotherapy treatment, but it’s still early days yet regarding it being approved as a successful cancer-fighting treatment.

CAR T is the shortened name for chimeric antigen receptor T cell and is a new type of cancer treatment that involves removing a certain amount of T-cells from the patient’s blood. These cells are then altered in a lab and then infused back into the patient’s blood where they hunt down, attach to, and finally kill tumor cells. However, it’s not always possible to harvest T cells from leukemia patients as they lack enough healthy ones already and a lot of time and money is needed for it because each set of T cells is specific to the patient.

Source: NY Times

However, if gene-editing is used, much of this uncertainty is taken away. When T cells were taken from donor recipients, four changes were made. Using TALENS, researchers manipulated the cells so that they became universal and suitable to be used in any person. The two babies who took part in the trial had both been through treatments such as chemotherapy and stem cell transplants. But does this mean that it’s the combination of treatments that have cured these two patients or was it just the CAR T alone? Stephen Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, said, “There is a hint of efficacy but no proof. It would be great if it works, but that just hasn’t been shown yet.”

So, it’s clear more research is needed before it can be confirmed one way or the other if CAR T is strong enough on its own to cure patients or is it the combination of this and gene-editing that make it so successful. I guess, for now, we will just have to wait and see.

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