The biotech world just got one step closer to editing genes in humans.
On Tuesday (June 21), a panel from the US National Institutes of Health approved with one abstention a proposal from researchers at the University of Pennsylvania to use gene-editing technology on immune cells from cancer patients as part of their treatment. The trial will be funded by the Parker Institute for Cancer Immunotherapy, founded by former Facebook president and Napster co-creator Sean Parker.
According to the MIT Technology Review, Parker refers to the project the “Manhattan project for curing cancer with the immune system.”
For the clinical trial, researchers hope to take immune cells from 15 patients. Each has one type of three cancers. Once the cells are removed, they will be edited using a technology called CRISPR to reprogram them to attack the cancer cells when they are returned to the patient, according to STAT.
As Quartz has reported previously, CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, relies on a trick scientists stole from bacteria. In an effort to protect themselves from dangerous viruses, certain bacteria can isolate snippets of genetic material in viruses and cut them out. Scientists later mimicked this technique to work on specific genes and replace harmful mistakes in genetic code with working code instead.
In theory, CRISPR can fix all of the specific miscoded genes in an organism. However, if the CRISPR technology makes a mistake, it could be fatal. In 2015, Chinese researchers caused an uproar by editing unviable human embryos using CRISPR just to see if it would work—only a small fraction of them were successfully altered.
Although the clinical trial has cleared this first step of approval, it has a ways to go before it can be tested in patients. Researchers still need to receive approval from the US Food and Drug Administration and each of the medical centers where patients are being treated, which include Penn, the University of California, San Francisco, and the MD Anderson Cancer Center in Texas.