On Tuesday, a federal biosafety and ethics panel gave the go-ahead for the first ever clinical trial using the game-changing gene-editing technology Crispr-Cas9. Scientists at the University of Pennsylvania want to use the technique to genetically alter patients' immune cells to attack three different kinds of cancer. This is a very big deal: it signals that we are perhaps much closer than previously thought to using genome-editing to fight some of the world's most common diseases.
In an interesting twist, the research is being funded by Napster founder-turned-tech mogul Sean Parker. Parker announced earlier this year that his foundation is funding a $250 million project to "solve" cancer. It is perhaps not surprising that someone from Silicon Valley, home of the "move fast and break things" mindset, would push the research community to more rapidly deploy the revolutionary gene-editing technique in humans.
But it's not all systems go yet. Tuesday's NIH panel is not the only hurdle UPenn researchers have to surmount. The experiment still requires the okay from the Food and Drug Administration, as well as the three medical centers where the researchers plan to conduct the research. But so far, the response has been enthusiastic. Panelists unanimously approved the study—even despite the researchers' misspelling of Crispr and use of Comic Sans in their presentation.
Researchers hope to enroll 15 patients diagnosed with myeloma, melanoma, and sarcoma in the trial. The scientists plan to remove patients' T cells from their bodies and use Crispr to snip out two genes in the cells that they hope will allow them to more effectively attack and kill tumor cells. Via MIT Review:
[Sean] Parker, a one-time hacker whose resume includes a run-in with the FBI, has said he thinks of T cells as being “like little computers” that can be reprogrammed.
Previous studies have tried something similar with T cells using older genetic engineering methods, but they have for the most part only worked with blood cancers and the tumors frequently come back. By using Crispr, scientists theorize that they can edit out two T cell traits that weaken them when in the midst of attack. Significant, too, is that by using Crispr, scientists can target multiple genes at once, instead of just one.
“Our preliminary data suggests that we could improve the efficacy of these T cells if we use Crispr,” U Penn’s Dr. Carl June told the National Institute of Health’s Recombinant DNA Advisory Committee.
Some scientists have expressed skepticism about the study's potential "off-target" effects, meaning that Crispr misses its target and accidentally snips the wrong bit of gene. Though Crispr is often described as a way to genetically "cut-and-paste," its accuracy isn't always spot-on. Scientists are working on improving its aim, but in the meantime that means that while cutting the snippet of gene you intended, sometimes you also inadvertently cut something else. Here, though, the NIH and others felt that for cancer patients the risk is outweighed by the potential benefits.
Before the UPenn study came up for approval, it was long expected that the first human trial of Crispr would be a clinical trial slated for 2017 that plans to use the gene-editing technique to treat a rare form of blindness. The cancer trial, though, puts Crispr on a much bigger stage. The study will not only answer questions about how effective Crispr is to treat certain kinds of cancer, but how safe and useful Crispr may be in humans in general. Is it truly the powerful tool we hoped it would be, allowing us to simply cut and paste our way out of the world's most deadly diseases? Or will Crispr be a treatment that—like chemotherapy and other cancer treatments before it—has consequences that prevent it from being the end-all-be-all cure.
It will likely be several years before we have the answer to that question. But in the grand scheme of things, that's not all that far away.