US feds to review first human gene-editing trial

The University of Pennsylvania has beaten Google-backed biotech Editas in the race to be the first to formally propose a trial of the gene-editing technology CRISPR in humans. The National Institutes of Health disclosed it is to review the trial proposal, which involves using CRISPR to modify T-cells in order to better target certain cancers, at its Recombinant DNA Advisory committee (RAC) on Tuesday 21 June. Commentators had expected Editas to be the first in-human application of CRISPR gene editing technology to treat a rare form of blindness. In-human trials of other gene editing techniques, such as TALEN have previously been conducted, with some degree of success; most famously treating a toddler with leukemia last year.

The federal committee that monitors DNA experiments on humans will make its first judgment on a CRISPR case next week. The Recombinant DNA Advisory Committee is looking at a proposal from the University of Pennsylvania, which wants to use the gene editing technique. The plan is to harvest T-cells from cancer patients and re-program them to better fend-off cancer cells. Rather than pumping people full of debilitating drugs and hope that cancers die off, the idea is that our own immune systems can do a better job. But in order to make it work, the cells need to have certain built-in safety features shut off, hence the need for oversight.

This isn't the first time that gene editing has been used to treat a human patient, nor the first time that it's been used against cancer cells. Late last year, we told you about doctors in Britain that used a different gene editing procedure -- TALEN -- to create modified T-cells. These cells were then implanted in a one-year-old child with a terminal case of acute lymphoblastic leukemia. By contrast, UPenn will be using CRISPR-Cas9, a different process which has been tested using human DNA in China, to do the same job.

The committee will only rubber-stamp the experiment if it's certain that researchers are giving their work the proper level of respect. As Dr. Carrie Wolinetz, a director at the National Institutes of Health, explains, officials will be looking to ensure the procedure "reflects well-established scientific and ethical principles." One key concern is that the process will shut down a component of our body's own immune response, much like an immunosuppressant drug. Should the team make a mistake, however, the consequences could pose a new set of risks for those undergoing treatment. But, since we're at the bleeding edge of medicine, it's hard to tell how far-reaching the consequences are.