For the first time in the United States, scientists will be able to edit the genes of humans to make them better at fighting cancer.
The phase I safety study will be the first use of a relatively new gene editing technology in humans. It received approval from a National Institutes of Health (NIH) panel earlier this week.
The move opens up the opportunity for CRISPR technology to reprogram the genes of someone with cancer to attack tumor cells.
The trial will involve 18 people with cancer. Researchers will remove some of their T cells — a type of immune cell that can be lethal to tumors — and perform three separate edits.
Through those edits, the new T cells will be able to detect and target cancerous cells, remove barriers that would prevent detection and targeting, and prevent the cancer cells from disabling attacks against them.
In the proposed study, researchers want to use CRISPR gene editing technology to reprogram a person’s T cells to attack myeloma, melanoma, and sarcoma tumor cells.
CRISPR, which stands for clustered regularly interspaced short palindromic repeats, began ramping up in 2013 when researchers discovered they could use it to cut the genome in cells in specific areas of their choosing.
The trial will use the CRISPR/Cas9, which uses bacterial enzymes that have been shown to be effective in manipulating immune responses.
Besides opening up new avenues for gene editing, the technology took off because it’s cheap, quick, easy to use, and doesn’t require years of training.
Special kind of editing
While Tuesday’s decision from the Recombinant DNA Advisory Committee (RAC) is a move forward, the technology still needs the approval of the U.S. Food and Drug Administration (FDA), which oversees all clinical trials.
Carrie D. Wolinetz, Ph.D., associate director for science policy at the NIH, said considering the study meant changing how the RAC reviews gene transfer trials and new technologies, including the unknown risks they could pose.
“Researchers in the field of gene transfer are excited by the potential of utilizing CRISPR/Cas9 to repair or delete mutations that are involved in numerous human diseases in less time and at a lower cost than earlier gene editing systems,” she wrote in a blog entry.
The NIH has voiced concerns with editing the human genome in fear of unknown consequences. In these experiments, the genes that would be altered wouldn’t be inheritable and would be the end of the line in individual patients.
Last year, Dr. Francis S. Collins, Ph.D., director of the NIH, emphasized that while NIH funding will help advance gene editing, it will not fund any use of gene editing technologies in human embryos.
Funding from Silicon Valley
The facility encompasses more than 40 labs and six centers, including Stanford Medicine, the University of California, San Francisco, and major cancer research universities.
The center’s focus is treating cancer through immunotherapy, a line of treatment that boosts the immune system to fight the deadly disease.
For the first trial, Nature reports, the gene editing will be done at the University of Pennsylvania.
“We are at an inflection point in cancer research and now is the time to maximize immunotherapy’s unique potential to transform all cancers into manageable diseases, saving millions of lives,” Parker said in a press release. “We believe that the creation of a new funding and research model can overcome many of the obstacles that currently prevent research breakthroughs.”