US Trial Shows 3 Cancer Patients Had Their Genomes Altered Safely by CRISPR

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US scientists have succeeded in genetically editing the immune systems of three cancer patients using CRISPR, without creating any side effects, a first for the tool which is revolutionizing biomedical research.


The highly anticipated results from the first phase of a clinical trial were published in the journal Science on Thursday.

They represent a stepping stone that doesn’t yet prove CRISPR can be used to fight cancer. Indeed, one of the patients has since died and the disease has worsened in the other two – but the trial does show that the technique is non-toxic.

Researchers at the University of Pennsylvania (UPenn) removed T-cells from the patients’ blood and used CRISPR to delete genes from the cells that might interfere with the immune system’s ability to fight cancer.

They then used a virus to arm the T-cells to attack a protein typically found on cancer cells called NY-ESO-1, and infused the cells back into the patients.

Edward Stadtmauer, the study’s principal investigator, told AFP that T-cell therapy, in which a person’s own immune system is exploited to destroy tumors, had been a major breakthrough of the past decade, but « unfortunately, even with that technology there are so many patients who don’t respond. »

The idea of this work therefore is to combine the two cutting-edge approaches to make T-cells even more powerful.​

There may not have been major clinical results this time around, but « to me the import of this study is not the clinical results but the fact that we were able to feasibly do this very complex procedure, » added Stadtmauer.

© Agence France-Presse


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