First CRISPR clinical trial passes initial safety review
On June 21, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR–Cas9 to help augment cancer therapies that rely on engineering patient’s immune cells.
Although other forms of gene editing have already been used to treat disease in people, the CRISPR trial would break new ground by modifying three different sites in the genome at once, which has not been easy to do until now. The study has also grabbed attention because—as first reported by the MIT Technology Review—tech entrepreneur Sean Parker’s new $250 million Parker Institute for Cancer Immunotherapy will fund the trial.
“Cell therapies [for cancer] are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. Gene editing could improve such treatments and eliminate some of their vulnerabilities to cancer and the body’s immune system, he says.
The proposed 2-year CRISPR trial will treat 18 patients with several types of cancers, e.g. myeloma, sarcoma, or melanoma, who have stopped responding to existing treatments. Researchers will remove T cells from these patients and perform three CRISPR edits on them. One edit will insert a gene for a protein engineered to detect cancer cells and instruct the T cells to target them, and a second edit removes a natural T-cell protein that could interfere with this process. The third is defensive: it will remove the gene for a protein that identifies the T cells as immune cells and prevent the cancer cells from disabling them. The researchers will then infuse the edited cells back into the patient.
Although endorsement from the NIH panel is a big step, the researchers must now seek approval from their own institutions’ ethics boards and the U.S. Food and Drug Administration. Others are likely nipping at their heels. Many thought the Cambridge, Massachusetts–based biotech company Editas Medicine would conduct the first CRISPR clinical trial—it has announced plans to use CRISPR to treat an inherited eye disease in 2017—but NIH has not yet reviewed a proposal from the company.
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