Soylent News has carried multiple previous articles about CRISPR, the "search and replace" tool for editing DNA (here [soylentnews.org], here [soylentnews.org], and here [soylentnews.org]).
Now scientists at three sites that are members of the Parker Institute—UPenn, the University of San Francisco in California, and the University of Texas MD Anderson Cancer Center in Houston, have proposed a UPenn-led project to genetically modify T-cells to treat leukemia and other cancers. From an article by Jocelyn Kaiser in Science Magazine [sciencemag.org]:
[...] The proposed clinical trial, in which researchers would use CRISPR to engineer immune cells to fight cancer, won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health, a panel that has traditionally vetted the safety and ethics of gene therapy trials funded by the U.S. government and others.
“It’s an important new approach. We’re going to learn a lot from this. And hopefully it form the basis of new types of therapy,” says clinical oncologist Michael Atkins of Georgetown University in Washington, D.C., one of three RAC members who reviewed the protocol.
The 2-year trial will treat 18 people with myeloma, sarcoma, or melanoma, who have stopped responding to existing treatments, at three sites that are members of the Parker Institute—UPenn, the University of San Francisco in California, and the University of Texas MD Anderson Cancer Center in Houston. [Carl] June [of UPenn] pointed out to RAC that his team already has experience with gene editing. They have used a different technique, called zinc finger nucleases, to disrupt a gene on T cells that HIV uses to enter the cells. In a small trial, this strategy appeared to be safe and has shown promise for helping HIV patients. Those data suggest that CRISPR gene editing should be safe in humans, June said.
Although RAC endorsement 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 RAC has not yet reviewed a proposal from the company.