A research team at the West China Hospital has become the first to inject a person with cells that have been genetically modified with the revolutionary CRISPR-Cas9 technique. The modified cells were injected into a patient with aggressive lung cancer as part of a Phase 1 clinical trial and researchers report the patient is doing well.
The news was reported in a Nature journal news story titled “CRISPR gene-editing tested in a person for the first time.”
This is not the first time that genetically modified cells have been inserted into human patients. A number of therapies, such as CAR T-cell therapy, rely on genetically altering a patient’s own immune cells, expanding them, and infusing them back into the patient. But researchers use viral and non-viral gene transfer systems to modify the cells.
The CRISPR-Cas9 technology has generated a lot of excitement in the scientific community because it is much simpler and efficient than any of the currently used methods. The system, which was first discovered in microbes, relies on two components — an enzyme (the Cas9 part) that cuts the DNA, and a molecular guide that can be programmed by researchers to tell the enzyme exactly where to cut. This allows researchers to efficiently remove or add new strands of DNA to the genome of a particular organism.
In this study, investigators isolated immune cells from the patient’s own blood and used the CRISPR-Cas9 technology to remove the PD-1 protein. This protein is part of an immune checkpoint that often is exploited by cancer cells to prevent immune cells from recognizing them, and researchers believe that removing this protein from immune cells could enhance the patients’ anti-tumor immune responses.
The patient at West China Hospital, who is about to receive a second injection, is the first of 10 patients expected to be enrolled in this Phase 1 trial, designed to determine the safety of two, three or four injections of CRISPR-Cas9 edited immune cells. Patients will be followed for six months to assess whether the injections are causing treatment-related adverse events, and to collect data regarding effectiveness of the treatment technique.
Similar clinical trials will soon start in the United States and China to assess the feasibility of using CRISPR-Cas9 edited immune cells to treat cancer patients. “I think this is going to trigger ‘Sputnik 2.0,’ a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” Carl June, who specializes in immunotherapy at the University of Pennsylvania in Philadelphia, said in the Nature article.