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In U.S. First, Scientists Safely Edit Cancer Patients' Cells With CRISPR

On Wednesday, U.S. scientists announced that they used the gene-editing tool CRISPR to modify the cells of cancer patients — a first in the United States — and that so far, the technique appears to be safe. It's too early to tell whether the edited cells will help patients live longer, but researchers think the approach looks promising and is an important first step for using CRISPR against other diseases.

There's an incredible amount of optimism around using CRISPR to treat or possibly cure a wide range of genetic disorders and cancers by snipping out detrimental strands of DNA from cells. The technology has drawn the attention of high-profile investors, including billionaire Sean Parker, co-founder of Napster and an early investor in Facebook, who helped fund the study. The idea is that CRISPR could eliminate disease at its source, rather than just alleviating symptoms, as the vast majority of pharmaceuticals on the market do. But CRISPR is a relatively new tool, and it is unclear how safe it is for human treatment. While experiments in animal subjects have looked promising, this new research gives scientists the first glimpse of how CRISPR will perform in people.

So far three cancer patients in the United States have received the experimental CRISPR treatment, which involved extracting certain immune cells — called T cells — from the blood, editing them in the lab, and infusing them back into the body. T cells are the body's natural mechanism for fighting infections and other foreign invaders, including tumors. But the reason cancer can persist in the body is because it can hide from the immune system, going undetected by these T cells. In this trailblazing study, the first proposed use of CRISPR in humans, researchers used CRISPR to supercharge patients' T cells by removing three genes that interfere with the immune cells' ability to bind to cancer cells and kill them.

Plans for this study were first reported in 2016 when an advisory group at the U.S. National Institutes of Health gave researchers at the University of Pennsylvania the green light. The first two patients were treated this April, and ultimately the study is slated to include 18 total patients with multiple myeloma, sarcoma, or melanoma whose cancers have relapsed or not responded to traditional cancer treatments, like chemotherapy or radiation.

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