First Cancer Trial Using CRISPR-Edited Cells to Begin Soon in China

According to a news report in Nature, scientists at Sichuan University’s West China Hospital are on track to initiate a clinical trial that will administer CRISPR-Cas9—edited immune cells in patients diagnosed with lung cancer.

According to a news report in Nature, scientists at Sichuan University’s West China Hospital are on track to initiate a clinical trial that will administer CRISPR-Cas9—edited immune cells in patients diagnosed with lung cancer. The hospital’s review board gave the team a go-ahead earlier this month.

Concurrent efforts are ongoing in the United States and just last month, and advisory panel at the National Institutes of Health approved a safety trial that has received the backing of Sean Parker. The cells are to be engineered at the University of Pennsylvania. The process will involve removing T cells from 18 patients with different types of cancers; CRISPR edits on the DNA of these cancers will:

  • Insert a gene to detect cancer cells
  • Delete a natural T-cell protein that inhibits the detection of cancer cells
  • Remove a protein that is recognized by cancer cells that can disable the T cells

The final product, treated as listed above, will then be reinfused back into the patient. The trial needs approval from the FDA and a university review board before it can start, which is anticipated to happen by year end.

According to Nature, the Chinese trial will enroll patients with advanced lung cancer who have failed on chemotherapy, radiation therapy, or other treatments. The plan is to isolate T cells from these patients to delete the gene that encodes the programmed death 1 (PD-1) protein, the checkpoint in T cells that prevents the body from setting up an immune response against cancer cells. This is the protein that is targeted by both nivolumab (Opdivo) and pembrolizumab (Keytruda). The US trial has a similar approach, according to the Nature report.

The PD-1 inhibitor antibodies have seen tremendous success and have been approved in a variety of cancer types, including melanoma, non-small cell lung cancer, renal cell cancer, and Hodgkin lymphoma.

Knocking down the gene in proliferating cells has the potential to be a much more powerful technique than antibodies, according to Timothy Chan, MD, an immunologist at the Memorial Sloan Kettering Cancer Center who spoke with Nature.

The key to this is validation of the cells—CRISPR is known to have errors that can insert edits in the wrong places on the genome. In China, a biotechnology company called MedGenCell is expected to validate the cells to ensure that the right genes have been knocked down, before the cells are reintroduced into patients.

“I hope we are the first. And more importantly, I hope we can get positive data from the trial,” said Lu You, an oncologist who’s leading the trial in China.