Targeted treatments: the rise of CAR T-cells

While broad-brush treatments like chemotherapy and radiation can destroy cancer cells effectively, damage to healthy cells is unavoidable. Our immune systems are programmed to fight off viruses and bacteria, but aren’t great at fighting off cancer, because it’s caused by our own cells multiplying out of control. Our immune system has difficulty recognising it as a threat and doesn’t attack it.

In the last decade, researchers have discovered a way to remove the cancer ‘blindfold’ on our immune systems and a new wave of targeted cancer treatment has arrived. These therapies are ‘locked on’ to cancer cells, concentrating their full power where it’s needed most. Damage to healthy tissue is minimised or nonexistent, vastly reducing the risk of side effects.

One promising technique involves modifying the genetic code inside the body’s own immune cells, enabling them to recognise, hunt down and destroy cancer cells. These modified cells are known as CAR T-cells.

GOSH researchers are leading the way in Europe in applying this incredible technology to acute lymphoblastic leukaemia (ALL) that has come back after treatment. In 2015, the first child in the world was treated with ‘universal’ CAR T-cells at GOSH and, in 2018, GOSH became the first hospital in the UK to

2013 Launch of one of the first European CAR T-cell studies, pioneered by Professor Persis Amrolia. It paves the way in this rapidly emerging field, but this type of CAR T-cells have now been superceded by ‘next generation’ cells that are more effective. 2015 GOSH immunologist Professor Waseem Qasim used CAR T-cells to treat a oneyear-old patient with ‘incurable’ leukaemia. His incredible world-first sparked a new wave of CAR T-cell research around the world.

treat patients with the licensed CAR-T therapy as part of standard care.

A new CAR T-cell therapy for relapsed ALL, developed by GOSH specialists Professor Persis Amrolia and Dr Sara Ghorashian, is being offered to children through a clinical trial known as CARPALL. Early results suggest this treatment could cause less side effects than existing CAR T-cell therapies, as well as potentially clearing the disease more effectively.

Now, researchers are working hard to improve CAR T-cell therapy, ensure it is available for all children who could benefit, and to apply it to other types of cancer.

Today

A new wave of targeted cancer treatment has arrived – CAR-T cell therapy.

2018 An 11-year-old boy at GOSH becomes the first NHS patient to receive CAR T-cell therapy. A landmark agreement between NHS England and the drug manufacturer means the drug (Kymriah) can now be offered to eligible NHS patients with relapsed ALL. 2019 Results of the CARPALL clinical trial at GOSH suggests a new CAR T-cell therapy has less side effects and could be more effective at treating ‘incurable’ ALL than similar treatments.