On Tuesday, the U.S. National Institutes of Health advisory panel approved the very first human clinical trial that will use the controversial and powerful gene-editing tool, CRISPR/Cas9.
In the clinical trial, which is being funded by technology billionaire Sean Parker's cancer institute — Parker Institute for Cancer Immunotherapy — the researchers will target three different types of cancer.
In particular, researchers from the University of Pennsylvania will use CRISPR/Cas9 to edit the human participants' T cells. The alternation is designed to make the immune cells more successful in fighting sarcoma, melanoma and multiple myeloma.
The researchers' proposal received a unanimous approval from the U.S. Recombinant DNA Advisory Committee with only one member who abstained. The team now needs to get approval from the U.S. Food and Drug Administration to proceed with the experiment.
Researcher Carl June and the team at the University of Pennsylvania developed the CAR T-cell treatment, which is a type of immune therapy wherein a patient's own T cells are procured, genetically edited and re-introduced into the bloodstream.
The scientists said that their objective is to create a new immunotherapy type using the gene-editing tool that will make the edited immune cells become more powerful and capable of surviving longer. This genetic modification will enable the immune cells to kill the malignant cells more effectively.
T cells are the white blood cells that play vital roles in eliminating malignancies, bacteria and other foreign invaders inside the body.
The researchers will use the CRISPR/Cas9 technology to make the T cells survive longer and lower the chances of the malignancies returning.
The experiment's initial trial will enroll over a dozen patients and will test the therapy's safety among humans. These experiments will be conducted at the University of Pennsylvania, the University of California, San Francisco and the MD Anderson Cancer Center.
Parker Institute for Cancer Immunotherapy CEO and President Jeff Bluestone said they are excited to be part of the country's first clinical effort to fuse the "two powerful therapeutic approaches" in treating cancer.
Last April, Parker made an announcement about the $250 million initiative to help fight cancer by bringing together researchers from across the United States who specializes in cancer immunology.
Since the announcement, the Parker Institute for Cancer Immunotherapy already has over 300 scientists who are working at 40 labs located in six institutions, namely the Stanford University, the University of California - Los Angeles, the University of California - San Francisco, the MD Anderson Cancer Center, the Memorial Sloan Kettering Cancer Center and the University of Pennsylvania.