CRISPR passes its first major cancer test and becomes the great hope to strengthen immune therapies

By active and passive. During the last years, we have not tired of repeating that CRISPR was an absolute technological revolution that opened the doors to things that less than a decade ago were inconceivable in genetics and biomedicine. The most paradigmatic example is perhaps cancer. On paper, the "genetic scalpel" could be used to help the patient's immune system attack cancer cells taking a (gigantic) step further the good results that T-cell therapies have been offering for years.

However, the truth is that everything was a great mystery. No one knew if CRISPR-Cas9 applications would be tolerated or could cause serious problems; nobody knew if the cells edited with these technologies would survive in the human body or tend to disappear. The news is that we already have an answer: a provisional, preliminary, and limited answer, but it is surely the best answer of the week.

A small step for CRISPR, but ...


And is that when a few months ago began to talk about this, all the experts called for calm. But data from the first North American CRISPR gene editing test in patients with advanced cancer shows that the patients experienced no negative side effects and that the modified T cells persisted in their bodies for months. The research is in phase 1, but everything suggests that genetic editing is a safe and feasible approach: CRISPR seems to be a very powerful tool to improve the natural ability of human immune cells to fight cancer.

Edward A. Stadtmauer and his team have selected three 60-year-old patients with advanced cancers who had not responded to standard treatments. First, they extracted T lymphocytes from the patients' blood and used CRISPR to strengthen their immune response capacity. Next, using a virus, the researchers "taught" T cells to attack a typical protein in cancer cells, NY-ESO-1. To finish, they again infused the cells into the patients and monitored their efficacy.

The good news, as I was saying, is that there were no toxicity related side effects and the modified T cells remained in the patients body until 9 months after treatment. The bad news is that there is still much to investigate. When I speak of a "provisional, preliminary and limited" answer, it is because, being a Phase 1 trial, "the big question that remains unanswered in this study is whether genetically modified T cells are effective against advanced cancer."

That will have to wait for future tests. However, what does seem clear is that "these findings offer the field of cellular engineering a guide to the safe production and non-immunogenic administration of genetically modified somatic cells." In other words, it lays the technological foundations for CRISPR to definitely start leaving laboratories and reaching clinics around the world.

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