The first Chinese CRISPR gene trial involving human participants will begin in August in Chengdu, at the West China Hospital, and it will target lung cancer patients.
The gene editing technique will include injecting modified cells in humans. The cells will be manipulated by using CRISPR-Cas9, which allows scientists to edit the DNA.
The researchers hope that the trial data will prove to be positive.
The Chinese CRISPR Clinical Trial
The participants in the clinical study will include patients with metastatic lung cancer and those for whom chemotherapy and medications showed no effects.
The team of researchers will extract T-cells from the patients’ blood and destroy a certain cancer-related gene. The gene will contain a protein called PD-1. The genetic editing will enhance the T-cell’s capacity to provoke an immune response while making sure that the healthy cells will be protected.
The T-cells will be modified in the laboratory and then multiplied so that later they could be injected into the bloodstream of the patients.
The purpose of this technique is to make the modified cells circulate into the blood stream and to make them target cancerous cells.
The Challenges of the Research Project
One of the risks of the research is that CRISPR modifications can lead to genome location errors, as the researchers could be modifying unwanted places in the DNA.
Therefore, each cell will be validated to make sure that the corrected genes are eliminated. The research team will work with Chengdu MedGencell, a biotechnology company, to verify each cell.
Another danger is that the modified cells will create a stronger immune response and target healthy cells, such as the ones in the gut or the adrenaline glands. The leading researcher explains that all the T-cells will be activated, which will turn into a mad race inside the body against anything that will be viewed as an anomaly.
One way to avoid this situation is to use the T-cells that appear inside the tumor, as they might be already specialized to work against cancerous cells. The main difficulty here would be that lung cancer tumors cannot be accessed so easy.
The clinical trial will use antibody therapies validated by the international scientific community, which would regulate the autoimmune response.
In the US, the researchers from the University of Pennsylvania led another clinical study on humans, but they used a different technique of gene editing. In this earlier study, they worked with HIV patients.
Now, they will be advising the Chinese team in their pursuit against lung cancer while working on their research project that will involve PD-1 and two other genes.
The American clinical trial will still have to wait until the FDA, and the US National Institutes of Health offer their approval.
Image Source: Wikipedia