First Trial to Use CRISPR in Humans Approved by Federal Biosafety and Ethics Panel

A federal biosafety and ethics panel approved the first-in-human study of the genome-editing technology CRISPR-Cas9 to create genetically altered immune cells to attack three kinds of cancer.

The early-stage clinical trial was proposed by scientists at the University of Pennsylvania with plans to enroll 15 patients with multiple myeloma, melanoma, and sarcoma to gauge the safety of the experimental therapy and test the feasibility of manufacturing genetically engineered and CRISPR T cells. The scientists plan to use CRISPR to genetically modify the T cells so that, when infused back into a patient, they can target and destroy tumor cells. Specifically, this trial is targeting PD-1 and TCR.

The trial would be conducted at the University of Texas MD Anderson Cancer Center, the University of California, San Francisco, and the University of Pennsylvania, pending further approval from the other medical centers and the U.S. Food and Drug Administration.

Source: Science, June 21, 2016.