Three cancer patients in the US have had their immune systems successfully genetically edited using CRISPR.
Scientists from the University of Pennsylvania removed the T-cells from the patients and used CRISPR to delete the genes that might interfere with their ability to fight cancer.
They then used a virus to help the T-cells attack a protein found on cancers cells called NY-ESO-1, and infused the cells back into the patients.
This major breakthrough was part of the Phase 1 trial, therefore the researchers focused on the safety of the treatment, whether the patients had side-effects, not its efficacy.
There may not have been major clinical results this time around, but “to me, the importance of this study is not the clinical results but the fact that we were able to feasibly do this very complex procedure,” Edward Stadtmauer, the study’s principal investigator, told AFP.
While this is still early days for CRISPR cells fighting cancer, finding out whether it will cause harm is the first step.
“Until now, it has been unknown whether CRISPR-Cas9-edited T cells would be tolerated and thrive once reinfused into a human,” CRISPR inventor Jennifer Doudna wrote, adding that the UPenn team’s findings “represent an important advance in the therapeutic application of gene editing and highlight the potential to accelerate development of cell-based therapies.”