Update on CRISPR: Closer to human trials?

Remember when we talked about CRISPR a little over a month ago? And we dreamt of the positive impact it could have on humans in the future? Well, dream no more–that day may be much sooner than we expected.

The NIH’s Recombinant DNA Advisory Committee panel, a federal biosafety and ethics panel, on Tuesday, June 21st, unanimously approved the first study in patients of the genome-editing technology CRISPR-Cas9. This experiment would use CRISPR to create genetically altered immune cells to attack three kinds of cancer in 15 patients.

Initially, as stated in our previous blog post, it seemed as though Editas Medicine in Cambridge, MA would be the first to use CRISPR in humans in an effort to treat a very rare eye disease called  Leber congenital amaurosis, which affects the light-receiving cells of the retina. But it seems as though researchers at University of Pennsylvania might beat them to it.

Researchers at the University of Pennsylvania proposed this experiment, which still needs to be approved by the medical centers where it will be conducted, as well as the Food and Drug Administration (FDA). If the study gets the green light from both, it would enroll patients with multiple myeloma, melanoma, and sarcoma, and be funded by the Parker Institute for Cancer Immunotherapy, which was launched this year by tech mogul Sean Parker (does Napster sound familiar?).

The trial will have 15 patients and will gauge the safety of this revolutionary therapy as well as see how feasible it will be on a larger scale. What exactly is being done in this study? The scientists would remove T cells, which normally target cells that are “foreign,” like bacteria, from patients with multiple myeloma, melanoma, or sarcoma. They would then use CRISPR to genetically modify the T cells so that, infused back into a patient, they can target and destroy tumor cells. In doing so the cells will be edited to remove two genes, one of which–PD-1–is a “key off switch of the body’s immune response.” By removing the gene, T cells may be able to overcome the ability of tumors to evade detection.

The trial, upon approval from the medical centers and the FDA, will be conducted at MD Anderson Cancer Center (nine patients) in Texas, and the University of California, San Francisco (three), as well as University of Pennsylvania (three), for a total of 15 patients.

Stay tuned for more updates on this–we’re watching history in the making!

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