A National Institutes of Health advisory panel on Tuesday approved the first human use of the gene-editing technology CRISPR, for a study designed to target three types of cancer and funded by tech billionaire Sean Parker’s new cancer institute.
The experiment, proposed by researchers at the University of Pennsylvania, would use CRISPR-Cas9 technology to modify patients’ own T cells to make them more effective in attacking melanoma, multiple myeloma and sarcoma.
The federal Recombinant DNA Advisory Committee approved the Penn proposal unanimously, with one member abstaining. The experiment still must be approved by the Food and Drug Administration, which regulates clinical trials.
The early-stage trial, which will involve more than a dozen patients, will be designed to test the safety of the therapy. It will be conducted at three medical institutions — Penn, MD Anderson Cancer Center in Houston and the University of California at San Francisco.
The study involves T cells, which are white blood cells essential for ridding the body of bacteria, malignancies and other invaders. Carl June and other researchers at Penn have pioneered an immune therapy, called CAR T-cell treatment, in which the cells are extracted from a patient, genetically engineered and then re-injected into the bloodstream. While the treatment shows promise, “our efforts have been hampered because the infused T cells become exhausted and die or cease to function,” the scientists said in explaining their proposal.
Under the study approved Tuesday, CRISPR would be used to remove two genes in the T cells to make the treatment last longer and reduce the chance of the cancer’s returning.
“Our goal is to develop a new type of immunotherapy using gene-editing technology that will enable the engineered immune cells to be more potent, survive longer and thereby kill cancer cells more effectively,” the scientists said.
Jeff Bluestone, president and chief executive of the Parker Institute for Cancer Immunotherapy, said in a statement, “We’re excited to be part of the first clinical effort in the United States to combine these two powerful therapeutic approaches to treat a devastating disease like cancer.”
In April, Parker announced a $250 million effort to bring together researchers specializing in cancer immunology at institutions across the country.
More than 300 scientists working at 40 labs in six institutions — Stanford, the University of California at San Francisco, UCLA, Penn, MD Anderson Cancer Center and Memorial Sloan Kettering Cancer Center — have already signed on.
In a blog post last week, an NIH official hinted at the risk and rewards of using CRISPR to fight cancer. “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,” said Carrie Wolinetz, associate director of science policy at NIH. “While the application of new gene editing technologies in this field has great potential to improve human health, it is not without concerns.”