Scientists plan to modify multiple genes.
The experts of the Advisory Committee on recombinant DNA (Recombinant DNA Advisory Committee, RAC) approved the application of the University of Pennsylvania to conduct tests on human genetic modification by using CRISPR/Cas9. In the process of the experiment will be improved the immune cells that effectively fight cancer.
Scientists plan to modify multiple genes, which did not allow the early methods of modification of the genome. The researchers will provide cancer patients with immune cells — T-lymphocytes, which then will introduce the neutralized viruses. The latter serve as a kind of transport for delivery of the gene encoding the receptor protein NY-ESO-1. This protein are often present on the surface of cancer cells, and almost never on the membrane healthy.
CRISPR also made the gene PD-1, which, as shown, reduces the activity of lymphocytes. Modified cells are then returned into the patient, where they begin to actively attack the tumor.
In two years the research involved 18 people with various types of cancer: multiple myeloma, sarcoma and melanoma. Tumors should be resistant to existing treatments. The experiments are going to three research centers: University of Pennsylvania, University of California San Francisco and University of Houston.
Some of the members of the Commission, considering the application, expressed concern that the University of Pennsylvania has a financial interest in the success of the experiment, because it has patents on the use of modified T lymphocytes for cancer treatment. By the decision of experts, the University should clearly define its role in research, whether it simply put the immune cells or directly participate in the trials. In the end, the Committee unanimously approved the experiment.
Advisory Committee on recombinant DNA organization at the U.S. Department of health, which deals with the approval and recommendations for the development of methods for modification or synthesis of DNA.