CAR T-cells are becoming somewhat of a household name, but a new phase I trial is, for the first time, using CAR 'NKT' cells, featuring genetic modification of a different immune cell, natural killer cells. Natural killer T-cells have long been known to be important in mediating the immune system's response to cancer, targeting both tumor cells and cells infected with viruses.

The new therapy is being used to treat relapsed neuroblastoma, a childhood cancer that 1 out of 5 children still do not survive long-term. Although many children can be successfully treated, the prognosis for children who have relapsed after initial therapy is poor and many families find that they have exhausted all treatment options.

Now, a new phase I clinical trial run jointly between Baylor College of Medicine and Texas Children's Hospital, both in Houston is evaluating the brand new treatment approach for children with relapsed neuroblastoma. The CAR-NKT cell treatment CMD-501, developed by UK-based cellular therapy company Cell Medica is the first of its kind to ever be trialed in any disease.

The first preliminary results from the trial named GINAKIT2 were shown yesterday at the Annual Meeting of the American Society of Gene & Cell Therapy in Washington DC. The presentations described results from testing and safety evaluations of the therapy in mice before also talking about results from the first two patients enrolled on the phase I clinical trial.

"Both patients had widely metastatic disease that has failed multiple therapies and many treatment regimens. The first patient achieved a stable disease at a four-week follow-up and the second patient had two bony tumors, one of which has completely disappeared and the other which is responding," said Dr Andras Heczey, pediatric oncologist and Assistant Professor in the Department of Pediatrics at Baylor College of Medicine, one of the leaders of the trial.