22 July 2016
The first trial of CRISPR modified cells on human subjects has been approved by the advisory board of West China Hospital in Chengdu.
The team behind the world first trial plan to extract immune system T-cells from the blood of patients with non-small cell lung cancer who have not responded to any other treatments.
The cells will be modified using CRISPR Cas9 editing, switching off the PD-1 gene, before being replicated and reintroduced into the patient’s body. Normally PD-1 functions as a 'friend or foe' signal within the immune system, but cancer cells circumvent it. Switching the gene off will allow the patient’s cells to resume attacking the cancer.
The primary aim of this phase 1 trial is to assess safety. The fear is that modified T-Cells which are no longer able to identify the invader from the invaded, could result in an auto-immune disorder as the body goes to war with itself. The team hope to minimise the risk by starting small, a single subject and a low dose, before eventually testing the cells on up to ten patients.
Several immunotherapy treatments, both pharmacological and antibody-based have already been approved by regulatory bodies in Europe and the US; a CRISPR based therapeutic could also prove effective against selected cancers
In the US, a similar trial aiming to treat patients suffering from myeloma, sarcoma or melanoma is set to begin by the end of the year.