CAR-T cell therapy

One particular area of interest at GOSH is utilising the body’s own immune cells – T cells – to fight cancer.

T cells are specialised immune cells that patrol our body, seeking and clearing up cells that are infected, for example, with a virus. But cancer cells can go unnoticed as they often look very similar to healthy cells. To use T cells as a cancer treatment, we reprogramme them with a cancer ‘detector’ called chimeric antigen receptor, or CAR for short.

The CAR enables T cells to spot cancer cells and spring into action, directly destroying cancer cells and alerting other immune cells of the cancer threat.

Yuvan was the first person to receive Kymriah - a type of CAR-T cell therapy, in the NHS, to treat his leukaemia at GOSH when he was just 11 in 2010.

Yuvan receiving CAR-T cell therapy

I remember a few flashes of this time at GOSH, like talking to the consultants and some of the good news we received but it’s mostly a blur. To anyone else undergoing this kind of research treatment, I'd advise them to keep calm: Just let the hospital teams work their wonders and hang tight. I worked with the team in the Young Person's Advisory Group for Research to design a game to help children to understand more about leukaemia and treatments in a fun and interactive way. It's really important how the hospital involves children and young people in their research design.


While promising for some forms of childhood cancer, CAR–T cell therapy can lead to life– threatening side effects and the cancer often returns as the treated T cells disappear from the body. Furthermore, obtaining children’s cells and returning them requires harsh, invasive procedures that some children simply aren’t well enough for.

Our research is tackling these issues head on.

For example, in blood cancers like acute lymphoblastic leukaemia (ALL) Professor Persis Amrolia and his team, in a project part– funded by GOSH Charity, have developed a new CAR molecule that can bind more rapidly to the cancer cells and remove them, reducing side–effects and improving cancer symptoms.

They’re working out why CAR–T cells last longer for some patients and not others (important because loss of CAR–T cells means the treatment doesn’t work), how they can make CAR–T cells persist in the body and have found that a special group of T cells are essential for destroying cancer cells.

In parallel, Professor Waseem Qasim and his team are leading research that uses ‘off– the–shelf’ CAR–T cells that work for anyone, so children don’t need to go through all the harsh measures to remove their cells. In 2022, he led teams from GOSH and UCL GOS ICH to deliver the world's first use of base editing to treat any condition, and continues to explore how we can use cutting edge technologies to improve tr

Research at GOSH is also working to tackle cancers with solid tumours, particularly neuroblastoma, a cancer that comes from the nervous system outside the brain.

A team led by Dr Karin Straathof, part–funded by GOSH Charity, developed a special CAR that instructs T cells to recognise a protein present only on the surface of neuroblastoma cancer cells. They’ve shown this works, leaving healthy cells untouched, and now plan to start a clinical trial of CAR–T cell therapy for children with neuroblastoma who aren’t responding to standard treatments.

GOSH teams are also now working towards developing CAR–T cell therapy for tumours inside the brain, as part of a clinical trial.