NIH panel approves CRISPR gene editing test in humans

The two-year trial will enroll 18 patients with one of three types of cancer who have not responded to standard therapy.

By Stephen Feller

WASHINGTON, June 23 (UPI) -- An advisory committee at the National Institutes of Health approved the first human trial to use gene editing as a method for fighting cancer by altering a patients' immune cells to more effectively battle tumor cells.

Researchers at three university medical centers plan to enroll and treat 18 patients using CRISPR/Cas9 technology for treatment of three types of cancer, according to Science Magazine.


The two-year trial is expected to test the safety of using CRISPR to edit three genes in patients' T-cells, which has not been possible before, which is expected to allow the immune system to fight cancer.

The trial is being funded by Napster co-founder and technology investor Sean Parker, who announced plans in April to fund the Parker Institute for Cancer Immunotherapy with $250 million as part of a larger effort to fund next-generation cancer treatments.

The CRISPR trial approved this week is one of the first expected to be funded among 40 university research programs Parker is partnering with to enhance and speed cancer research, the Washington Post reported.

"Researchers in the field of gene transfer are excited by the potential of utilizing CRISPR/Cas9 to repair or delete mutations that are involved in numerous human diseases in less time and at a lower cost than earlier gene editing systems," Dr. Carrie Wolinetz, associate director for science policy at the NIH, wrote in a blog post. "While the application of new gene editing technologies in this field has great potential to improve human health, it is not without concerns."


For the trial, researchers at the University of Pennsylvania, University of California San Francisco and the University of Texas MD Anderson Cancer Center will treat 18 patients with myeloma, sarcoma or melanoma whose cancer is not responding to standard treatments.

The scientists will remove T cells from patients and, using the harmless virus that powers CRISPR, will give the cells a receptor for the NY-ESO-1 protein found on certain tumor cells so they can attack them.

A gene for the protein PD-1, which other forms of cancer immunotherapy aim at as well, will be targeted to disrupt its ability to help cancer cells hide from immune system T cells. On top of this, two genes will be knocked out to help make the treatment more effective.

The researchers plan to watch for immune reactions to the Cas9 enzyme, which instigates the changes to DNA, as well as watching for changes made in the wrong part of a gene, which could potentially create or trigger cancer genes -- though previous testing showed this happened just once in a test run of the method.

"It's an important new approach," said Michael Adkins, a clinical clinical oncologist at Georgetown University and one of three RAC members who reviewed the protocol. "We're going to learn a lot from this. And hopefully it will form the basis of new types of therapy."


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