A new test by scientists in China aims to advance the gene-editing process in order to ultimately help patients fight deadly diseases.
Using CRISPR-Cas9 gene editing, Lu You, an oncologist at West China Hospital at Sichuan University, will test the gene editing treatment on lung cancer patients.
The technique involves using CRISPR to alter the patients’ genes. In the process, Dr. You will take the patients’ T-cells from their blood and use CRISPR to locate genetic sequences within a chromosome. They will remove a piece of the chromosome and replace it with another. This gene is responsible for encoding a protein called PD-1. It is this protein that allows cancer cells to avoid detection by the immune system.
The biotechnology company Chengdu MedGenCell, which is collaborating on the project, will examine the genes to ensure they were edited correctly. From there, the altered genes will be multiplied and injected into the patients. If the test is successful, the immune cells will attack the cancer cells.
The other critical portion of the test is to make sure the immune cells do not attack other parts of the body, causing additional problems. If successful, this gene-editing treatment could open the door to revolutionary treatment in fighting cancer and other diseases with limited treatment options.
Patients in the trial
The ten patients selected for the study have tried multiple standard treatments for mestastatic, non-small-cell lung cancer without success.
Because this is the first human trial with CRISPR and there are some significant risks in not knowing how the immune cells will react, Dr. You is taking special precautions. A single patient will be treated first to make sure there are not any problems before treating the other patients.
A similar clinical trial involving CRISPR could occur in the United States later this year, but such trials require FDA 510K clearance submission. Such trials can be fast-tracked with a third party review program such as TPRG.
Advances in China
If Dr. You’s trial is successful, it could lead to additional trials and give China a jump start in the world of gene editing treatments. Regardless of the study’s success, it is likely to provide valuable information on the gene editing process and CRISPR, allowing future studies to build on the work of that done by Dr. You.