CAR-T cells for treatment of neuroblastoma

Overview

Treating refractory and relapsed neuroblastoma remains a huge challenge, with survival rates as low as 10% for patients with this disease. Standard therapies which use specific drugs to kill the cancer cells are typically ineffective in these cases, but newer studies have shown that optimising the patient's immune system to kill the cancer cells is more promising.

In refractory leukaemia, patients who have previously been considered as incurable have been successfully treated using CAR-T cells. These are genetically engineered T-cells from the patient's own immune system that are reintroduced to the patient to efficiently attack a target cell. Early studies have developed CAR-T cells that are effective at killing neuroblastoma cells in relapsed/ refractory cases, but more work is needed to make them work over a long period of time.

The study led by Prof John Anderson will spend two years refining the CAR-T cells to decrease toxicity to the patient and investigate combination drugs, which will help them to last longer in the body. Specifically, this will be achieved by:

• Screening available drugs that will overcome the immune suppression that comes from neuroblastoma tumours.
• Optimise the CAR-T cell engineering so that it more precisely targets neuroblastoma cells, therefore reducing toxicity to the rest of the body.

Impact

If the study is successful in engineering more efficient CAR-T cells, as well as an effective combination therapy that incorporates them, it will become feasible that this treatment could be introduced into an existing clinical trial as an amendment. This would increase the treatment options available for relapsed/ refractory patients.