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Two Tweaks and a Treatment

A federal safety panel has approved a plan to use the genome-editing tool CRISPR/Cas9 to alter patients' immune cells as part of a treatment approach for three cancer types, as GenomeWeb has reported.

University of Pennsylvania researchers had proposed editing two genes — PD-1 and TCR — within the T cells of patients with myeloma, melanoma, or sarcoma to bolster patients' immune response to cancer. Those altered cells would then be infused back into patients.

"Our preliminary data suggests that we could improve the efficacy of these T cells if we use CRISPR," UPenn's Carl June told the National Institute of Health's Recombinant DNA Advisory Committee, according to Stat News.

The committee was unanimous in its decision to approve the trial, and most committee members were enthusiastic about its prospects. University of Southern California's Paula Cannon called it "innovative" and Michael Atkins Georgetown University School of Medicine said it was a really exciting first-in-human "study that could hopefully form the basis of new [cancer] therapies," Stat News reports.

If it also receives institutional approval and a green light from the Food and Drug Administration, the clinical trial will start with 15 patients to determine the approach's safety and feasibility.