CRISPR-Edited T Cells Used in Cancer Patients for the First Time in the US
Preliminary research suggests that using CRISPR to treat cancer is safe in humans and could become a feasible therapeutic method in the future, although its efficacy is still unknown. Results from an ongoing clinical trial, led by hematologist Edward Stadtmauer of the University of Pennsylvania Perelman School of Medicine, will be presented at the American Society of Hematology meeting in December. The presentation abstract was published online yesterday (November 5).
Stadtmauer's team procured T cells from three cancer patients—two with multiple myeloma and one with sarcoma—through a blood draw, and genetically modified the cells' DNA using CRISPR. They inserted a gene from a virus into the immune cells that cause the cells to target the protein NY-ESO-1, found on cancer cells, and deactivated three genes within the cells that could interfere with their cancer-fighting ability. Two of the gene edits inactivate TCRα and TCRβ, causing T cell receptors to be removed. Without the receptors, the cells can more easily bind to cancer cells. The third edit disables PDCD1, a gene that can kill T cells.
The researchers then infused the cells back into the patients, who have since had no significant adverse effects after six months, according to NPR. Research is still underway to determine whether the altered T cells are having any effect on the cancers, according to The New York Times.
"This trial is primarily concerned with three questions: can we edit T cells in this specific way? Are the resulting T cells functional? And are these cells safe to infuse into a patient? This early data suggests that the answer to all three questions maybe yes," says Stadtmauer in a news release. The team is planning to conduct a clinical trial with 18 patients in the future.
"I' m just so excited about this,"Jennifer Doudna, a biochemist at the University of California, Berkeley, who helped develop CRISPR techniques and was not involved in the study, tells NPR. “It's an important step on the path toward using CRISPR-Cas genome editing in patients and shows the potential of this technology to be a safe and effective therapy.”
Correction (November 6): A previous version of this article incorrectly stated that this trial marks the first time CRISPR-edited T cells have been used in cancer patients. In February 2018, NPR reported that Chinese researchers used CRISPR to edit T cells in order to treat cancer. The Scientist regrets the error.
Keywords: cancer clinical research, CRISPR, disease & medicine, gene editing
Ubigene Biosciences is co-founded by biological academics and elites from China, the United States, and France. We are located in Guangzhou Science City, which serves as a global center for high technology and innovation. Ubigene Biosciences has 1000㎡ office areas and laboratories, involving genome editing, cell biology technology, and zebrafish research. We provide products and services for plasmids, viruses, cells, and zebrafish. We aim to provide customers with better gene-editing tools for cell or animal research.
We developed CRISPR-U™ and CRISPR-B™(based on CRISPR/Cas9 technology) which is more efficient than general CRISPR/Cas9 in double-strand breaking, CRISPR-U™ and CRISPR-B™ can greatly improve the efficiency of homologous recombination, easily achieve knockout (KO), point mutation (PM) and knockin (KI) in vitro and in vivo.
Genome Editing Platform
——Focusing on the Application of CRISPR-U™ and CRISPR-B™ Gene Editing Technology
Cell Biology Platform
——Focusing on primary cell
2. Provides culture strategies and related products for different cell types.3. Provides cell biology-related services such as cell isolation, extraction and validation.