CRISPR-Cas9 Human trial in China

It was announced today that CRISPR/Cas9 was used for the first time in a human clinical trial for lung cancer.

CRISPR/Cas9 is an exciting, relatively new, technique used for editing DNA. This technology holds promises for curing numerous genetic disorders and various types of cancers. While there have been experiments performed in mice, there have been no reports of CRISPR being used in humans.

Oncologist Lu You at Sichuan University in Chengdu delivered modified cells to the patient on 28 October. However, China is not the only country working on using CRISPR to treat cancer. A clinical trial in the US is slated to start in early 2017.

For this particular trial, a protein (PD-1) was disabled in immune cells. When PD-1 is active it causes the immunes cells to not fight as hard. By disabling the protein the group hopes the immune cells will be more aggressive and attack the cancer cells. In total, the group plans to enroll 10 people and give each person 2-4 injections. While the group hopes to see benefits from the treatment, this trial is primarily to test safety.

Cancer-fighting strategies that target PD-1 have been used successfully in the past. However, using replicating cells brings new challenges. Proper dosing could potentially be an issue, over-activation of the immune system could lead to an auto-immune response. In addition, this technique requires that cells from patients be removed, edited, and multiplied. This takes time and cost money. It is unknown whether the gains will be worth the added effort.

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