First Human Trial Using CRISPR Gene-Editing in US Begins

The first ever human trial using CRISPR gene-editing in the US has reportedly begun.
John Loeffler

The first US medical trial of CRISPR gene-editing in humans has reportedly begun, testing the effectiveness of the technique in fighting cancer.

First Human Trials for CRISPR in US Underway

According to a report by NPR, the first US human trial of CRISPR gene-editing as a treatment for cancer has begun, making a major step towards bringing the powerful gene-editing tool to practical use in a medical context.


CRISPR has been used successfully in animal and plant species in the US, but it has never been used in human trials before. Now, a study by the University of Pennsylvania in Philadelphia has begun to determine the efficacy of CRISPR gene-editing in humans afflicted with treatment-resistant cancers.

So far, according to a university spokesperson, two patients have been treated using the technique, which makes targeted alterations to specific genes in a person’s DNA.

The treatment involves taking immune system T-cells that the body uses to fight cancer, modify their genes in a lab, and reintroduce the new edited T-cells into the patient. The goal is to circumvent the kinds of tricks that cancer cells use to evade or otherwise thwart their destruction by the immune system.

So far, the results of the treatment are not known as the trial is still in the very early stages and the trial has been approved for 18 patients overall. "Findings from this research study will be shared at an appropriate time via medical meeting presentation or peer-reviewed publication," a university spokesperson said in an email to NPR.

Trial the First of Several Planned for US, Canada, and Europe

This trial isn’t the first use of CRISPR in human trials, however. Studies of CRISPR gene-editing have been conducted in China, targeting cancer, and in Germany, targeting the blood disorder beta thalassemia.

There has also been the controversial case earlier this year of a Chinese doctor claiming to have used CRISPR to edit the genes of twin girls while they were still embryos, ostensibly to prevent the transmission of HIV from an HIV positive parent.

In western countries, several trials are set to begin soon targeting a variety of diseases, not just cancer.

"2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense," said gene-editing specialist Fyodor Urnov, from the Altius Institute for Biomedical Sciences in Seattle and the University of California at Berkeley.

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